Aspirin-induced asthma (AIA) is a condition where asthma symptoms can develop after taking aspirin or other nonsteroidal anti-inflammatory drugs (NSAIDs). Its also known as aspirin-exacerbated respiratory disease (AERD) or Samters Triad.

The American Academy of Allergy, Asthma, and Immunology (AAAAI) estimates that 9 percent of adults have asthma and that 30 percent of adults who have asthma and nasal polyps may also have AERD.

Read on to learn more about the underlying causes and risk factors of AIA as well as how this condition may be treated.

Acetylsalicylic acid (aspirin) is a type of NSAID used to relieve pain, inflammation, and fever. Similar medications include ibuprofen (Advil) and naproxen (Aleve).

Aspirin and other NSAIDs interact with an enzyme known as cyclooxygenase-1 (COX-1). While the exact triggers are unknown, its thought that people with AIA have a sensitivity to the way these medications inhibit this enzyme.

You may be more prone to AIA if you have all three of these conditions:

A doctor may still recommend aspirin for the treatment of other conditions, such as preventing heart attacks or strokes, in cases where a person may have already experienced one of these conditions and when the benefits outweigh the risks of triggering asthma symptoms.

Symptoms of AIA tend to develop shortly after taking aspirin or other NSAIDs often within minutes or hours after exposure.

While its important to address any suspected symptoms of AIA with a doctor, heres a breakdown of mild symptoms as well as more severe symptoms that require immediate medical attention.

Mild symptoms of AIA may include:

More severe symptoms of AIA can make it harder to breathe even if you take a rescue inhaler. Although rare, these acute symptoms can be life threatening.

Seek emergency medical help if you experience the following:

People who develop AIA are usually between ages 20 and 50 years old and likely have a combination of:

You may also be more susceptible if you experience the following on a recurring basis:

Age is another consideration. You more generally can become more vulnerable to side effects from NSAIDs as you age past your 50s.

Its also possible that reactions to aspirin could be induced by drug allergies. Besides NSAIDs, other common drug allergies include:

Symptoms of AIA may also be further exacerbated if you also drink alcohol. The AAAAI estimates that 75 percent of people with the condition may experience symptoms after drinking alcohol along with aspirin use.

AIA is typically diagnosed with the help of an asthma specialist, such as an allergist, pulmonologist, or immunologist.

Theres not just one test that can diagnose AIA. Instead, a diagnosis is made with a combination of the following factors:

A doctor may also recommend ordering a test called an aspirin challenge to rule out drug allergies. This involves taking aspirin either in the doctors office or at the hospital while under medical supervision. Any reactions you have to taking aspirin can then be identified and treated.

Along with avoiding NSAIDs, treatment for AIA involves managing symptoms of asthma, sinusitis, and nasal polyps.

You can also talk with a doctor about the following options.

Home treatments can include:

A doctor may recommend one or more of the following medical treatments:

Besides exacerbated asthma symptoms, complications of AIA may include hives (urticaria). The AAAAI estimates that between 20 and 40 percent of people who have chronic hives may have worsening symptoms if they also experience AIA. A type of swelling called angioedema can also occur.

Its also important to consider long-term side effects of taking aspirin and other NSAIDs, especially when taken for longer than recommended. These include:

Avoid mixing aspirin with the following, too:

Aspirin is a type of NSAID primarily used to relieve pain. But be careful using aspirin if you have a history of asthma, sinusitis, and nasal polyps. These underlying conditions may put you at a higher risk of developing AIA.

Talk with a doctor if youre concerned about the risks or side effects of taking NSAIDs or if you have a history of side effects after taking these types of medications. They can help diagnose and treat potential AIA along with related medical conditions.

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What Is Aspirin-Induced Asthma? Causes, Symptoms & More - Healthline

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Leading clinicians at Allegheny General Hospital support first islet cell transplant to treat chronic pancreatitis.

PITTSBURGH (PRWEB) October 05, 2021

Allegheny Health Network (AHN) today announced a groundbreaking new capability for treating patients who suffer from chronic pancreatitis, an inflammation of the pancreas that occurs over many years and, in severe cases, can be life-threatening. Every year, more than 80,000 people are diagnosed with the disease, according to the National Pancreas Foundation.

Surgeons at Allegheny General Hospital (AGH) have joined a select group around the country performing islet cell transplantation to restore the functions of a diseased pancreas. AGHs Institute of Cellular Therapeutics, is one of just a few in the nation that specializes in islet cell isolation, a highly sophisticated process in which islet cells, such as those that produce insulin, are extracted from the patients removed pancreas and transplanted back into the body. For the past six years, the institutes Islet Cell Isolation Laboratory has extracted and processed these life-saving cells to assist in the care of patients at select islet cell transplant centers around the country. With the launch of its own transplant program, AHN becomes one of just a few medical centers in the nation able to provide the comprehensive therapy from start to finish.

Pancreatic islets are tiny clusters of cells scattered throughout the pancreas. Included among these islets are beta cells, which produce the hormone insulin that helps the body absorb glucose from the bloodstream and use it for energy. Diabetes develops when the pancreas does not make enough insulin, the bodys cells do not effectively use insulin, or a combination of both. Massimo Trucco, MD, Director of AHNs Institute of Cellular Therapeutics and an internationally preeminent diabetes researcher, leads the islet cell extraction team at the hospital.

The islet cell transplant is critical for patients with immense pain and have failed other therapies. Dr. Trucco and his staff collaborate with AHNs gastroenterology, abdominal transplant, endocrinology, psychology and social services teams to complete the procedure and provide wrap-around patient support.

Chronic pancreatitis is a debilitating disease that can lead to frequent hospitalizations, higher use of narcotic pain medication and a lower quality of life, said Abhijit Kulkarni, MD, FASGE, an AHN gastroenterologist who evaluates patients for the islet cell transplant procedure. This treatment really represents the pinnacle of pain management for pancreatitis care and can be a true lifeline for the most critically ill patients.

According to Rita Bottino, PHD, from AHNs Institute of Cellular Therapeutics, the poor condition of the pancreas can make extraction of islet cells challenging. We sometimes have to be a little creative in finding a way to inject enzymes into the organ that will break the matrix that holds the cells together. By injecting enzymes, clusters of cells or single cells will be released from the organ and those are the kind of insulin-producing cells that we ultimately want to transplant back to the patient.

Once the islet cells are extracted and ready for transplant, AHN surgeons Harry Williams, MD, Ngoc Thai, MD, PhD, and Tadahiro Uemura, MD, PhD, transplant the cells into the patients own liver through the portal vein. Called an autologous islet cell transplant (TPAIT), the cells continue to produce insulin to control blood sugar levels in the body, eliminating the risk of becoming diabetic.

So we remove the diseased organ, which is causing debilitating symptoms for the patient, while creating a new pathway for insulin production in the body. And the advantage of a smaller time interval between extraction and transplant of these cells is significant and we believe will result in even better outcomes for our patients, said Dr. Williams.

Thus far, AHN has completed one TPAIT procedure and prepared more than 100 islets for regional hospitals including UPMC and The Cleveland Clinic. In addition to treating patients, AHN supplies research donor islets to Mt. Sinai Hospital, Stanford University Medical Center, Vanderbilt University Medical Center, and the universities of Pennsylvania, Miami and San Francisco, among others.

Our ultimate goal when starting our islet cell isolation lab several years ago was to ultimately develop the transplant capabilities and become one of the few one stop shops for this highly specialized care in the country, said Dr. Thai, Director of AHNs Center for Abdominal Transplantation. Having some of the worlds foremost experts in this field at our institution, like Dr. Trucco and his team, has afforded us with an extraordinary opportunity to build an internationally leading program.

To learn more about AHNs Cellular Therapeutics Institute or Transplant program, please visit http://www.AHN.org.

About the Allegheny Health Network: Allegheny Health Network (AHN.org), a Highmark Health company, is an integrated healthcare delivery system serving the greater Western Pennsylvania region. The Network is composed of 13 hospitals, ambulatory surgery centers, Health + Wellness Pavilions, an employed physician organization, home and community based health services, a research institute, and a group purchasing organization. The Network provides patients with access to a complete spectrum of advanced medical services, including nationally recognized programs for primary and emergency care, trauma care, cardiovascular disease, organ transplantation, cancer care, orthopedic surgery, neurology and neurosurgery, womens health, diabetes, autoimmune disease and more. AHN employs approximately 21,000 people, has more than 2,500 physicians on its medical staff and serves as a clinical campus for Drexel University College of Medicine and the Lake Erie College of Osteopathic Medicine.

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AHN Performs Its First Islet Cell Transplants to Treat Chronic Pancreatitis - PR Web

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Otto Warburg probably would have been sent to a concentration camp if the Nazis werent hoping he could cure cancer.

Warburg was a Jewish gay man living openly in Berlin with his partner as Hitler rose to power. Warburg was also a biochemist, as brilliant as he was arrogant. In the 1920s, he discovered a hallmark of cancer, now called the Warburg effect. Malignant cells are ravenous for glucose, or blood sugar, consuming 10 times more than healthy cells. He dedicated his career to studying this strange metabolic anomaly because he believed it was the root cause of cancer.

He won the 1931 Nobel Prize for his work. But it was mostly forgotten by the 1950s, eclipsed by the molecular genetics revolution that set off a search for mutated, cancer-causing genes. He died in 1970 at age 86.

The rise, fall, and recent resurgence of research into cellular metabolism is the subject of Ravenous: Otto Warburg, the Nazis, and the Search for the Cancer-Diet Connection. Author Sam Apple, a journalist based in the Philadelphia suburb of Wyndmoor, weaves together this complex narrative in a way that makes arcane science accessible and fascinating.

The book is also thought-provoking for anyone interested in avoiding cancer and who isnt?

Its not obvious to me that there would be a breakthrough cancer therapy if Warburgs focus on cellular metabolism had not been shunted aside, Apple said in a recent interview. More likely, there would have been more attention to the relationship between our diets and the metabolism of the whole body and cancer. I think this could have had a dramatic impact on cancer prevention. But the link is still is not widely appreciated.

Otto Warburg was related to a wealthy German Jewish clan of bankers, scholars, and influencers. He was groomed for scientific greatness by his father, Emil Warburg, a leading physicist of the time.

The pinnacle of Otto Warburgs career happened to coincide with the rise of the Third Reich. When Warburg refused to sign a declaration of Aryan descent, the Nazi customs official tasked with asking Warburg to lie endeavored to get him punished by the Kaiser Wilhelm Society, the parent organization of Warburgs scientific institute.

Warburg not only managed to get off scot free, but he asked the Wilhelm Society president to ask the Reich Ministry of Finance to rewrite racial decrees so that non-Aryan institute directors would be treated like Aryan directors.

In 1934, at a moment when Hitler had already begun sending Germans to concentration camps, Otto Warburg, a gay man of Jewish descent, wanted Nazi laws rewritten according to his personal needs, Apple writes in the book.

As the author explains, cancer rates were inexplicably rising in Germany and other developed countries, and the Nazis fear of the disease ran almost as deep as their antisemitism and homophobia They despised Warburg, but needed his scientific genius.

Warburg invented several new tools to study the metabolism of cells, including the manometer, an instrument used to measure the force exerted by a gas or liquid.

He knew that in a healthy cell, blood glucose was normally converted to energy in a process using oxygen. This process, which involves enzymes that Warburg spent years identifying, occurs in the cells power stations, now known as mitochondria.

He discovered, to his amazement, that cancer cells broke all the metabolic norms. In addition to overconsuming glucose, the cells turned it into energy using an inefficient process that did not require oxygen even though plenty of oxygen was available.

The cancer cells were chopping glucose molecules in half and spitting the fragments right back out of the cell, Apple writes in an elegantly simple description of anaerobic glycolysis, or fermentation, the biochemical process that also gives us beer and wine. Cancer cells, Warburg realized, were fermenting glucose just as simple organisms like yeast and bacteria do.

But why? Warburg hypothesized more like proclaimed that cancer cells mitochondria were somehow defective, so the cells had to resort to a backup power generator, namely fermentation.

Today, while the search for answers continues, the evidence suggests that fermentation is not a response to a defect. Rather, it gives malignant cells an advantage as they turn into uncontrollable, immortal renegades.

Glucose molecules are the building blocks that cancer needs to create daughter cells, Apple boiled it down during the interview. The most influential scientists now think its about the cancer cells bioenergetic needs.

Following the 1953 discovery of the structure of DNA the genetic instructions for everything cells do oncology researchers became focused on finding and fixing the defective genes that give rise to cancer.

In that postwar era, Warburgs focus on cell metabolism was seen as outmoded, like studying the fuel line in hopes of understanding a high-tech engine.

Beginning in the 1990s, however, some leading researchers realized that certain cancer-causing genes known for their role in cell division also regulated cells glucose consumption. One of those scientists, Chi Van Dang, former director of the University of Pennsylvanias Abramson Cancer Center and now scientific director at the Ludwig Institute for Cancer Research, showed that MYC, a family of genes regulating cell proliferation, also targets an enzyme that can turn on the Warburg effect.

Metabolism-centered cancer therapies that effectively starve tumors are no longer just a concept. Two drugs, ivosidenib and vorasidenib, have already been approved by the Food and Drug Administration for a form of leukemia and are now being tested in brain cancer patients. Rafael Pharmaceuticals experimental therapy, devimistat, has had impressive early results in bile duct cancer trials.

But as Apple points out, cancer is an incredibly persistent foe. It can mutate to evade chemotherapy, molecularly targeted therapies, and even newer immune-boosting therapies. The same thing may happen with metabolic therapies. Whats more, virtually all cancer treatments come with significant side effects.

Thats why the implications for preventing cancer in the first place are so important.

Population-wide studies have directly linked 13 cancers including breast, bladder, lung, colon, liver, and gynecological cancers to the same metabolic abnormalities that are driving the twin worldwide epidemics of obesity and diabetes. The most striking thing that the cancers, obesity and diabetes have in common is resistance to insulin, the vital hormone that enables cells to absorb blood glucose and turn it into energy. To compensate for this resistance, the pancreas pumps out more and more glucose.

The hypothesis is that patients high blood sugar impacts tumor growth by providing cancer cells with an abundance of the fuel they thrive on.

Apple spends almost half his book taking deep, incisive dives into research on the insulin connection. Refined sugar, which is a combination of glucose and fructose called sucrose, contributes to insulin resistance, So does fructose, or fruit sugar especially when it is concentrated in high-fructose corn syrup, the ubiquitous processed-food additive. Consuming fructose, a carbohydrate, also appears to make people add fat tissue more readily than actual fats, such as such as butter.

Precisely how much sugar is too much may be different for each person, depending on genes and age and exercise habits and capacity to store fat safely, he writes. But the path from refined sugar added to our diets to insulin resistance ... to cancer is now well understood and based on widely accepted science.

He touches only glancingly on a fundamental problem: Even if sugars role as a cancer-promoter becomes an article of faith, cutting back on it is tough. And controversy, not to mention quackery, abounds in the field of nutrition. The ketogenic diet, for example, restricts carbohydrates, which are converted to glucose in the body. The diet has shown promise in weight loss studies, as well as in relieving neurological disorders such as epilepsy. But the esteemed Mayo Clinic says the diets high level of saturated fats, combined with limits on nutrient-rich fruits, veggies and grains, is a concern for long-term heart health.

In the final chapter, Apple weaves in a chilling anecdote:

Sugar, of course, cannot be blamed for Nazism or for turning Hitler into a madman. But as his madness grew, so, too, did his taste for sweets. It wasnt only his cherished Viennese pastries. On any given day, Hitler might consume two full pounds of chocolates. He even added sugar to his wine.

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An old idea from a German Jewish scientist spared by the Nazis is getting new life: Prevent and treat cancer by cutting out sugar - The Philadelphia...

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INTRODUCTION Clustered regularly interspaced short palindromic repeats (CRISPR) are a family of DNA sequences, which constitute a primitive immune system that is responsible for protecting prokaryotic cells from phage infections.

New York, Aug. 24, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global Forecasts, 2021-2030" - https://www.reportlinker.com/p06130494/?utm_source=GNW It was first described in 1987, however, its potential as a gene editing tool was not realized until 2012. Since then, the CRISPR revolution has not shown any signs of slowing down and has been responsible for significant advances in molecular biology and therapy development. Fundamentally, the CRISPR/Cas system involves specific palindromic DNA sequences which work in tandem with a family of caspase enzymes (Cas9, Cas12), in order to excise gene fragments with high precision. Compared to the other targeted nuclease-based systems, CRISPR is relatively faster, and cost-efficient; as a result, the demand for this gene editing tool is very high. The relatively recent discovery / development of novel accompanying nucleases, namely Cas12a, Cas13, Cas14 and dCas9, has significantly improved the precision of this technology. Presently, there are several companies using different variants of the CRISPR/Cas technology for basic research, and the development of gene editing solutions. However, the therapeutic use of this versatile genetic manipulation tool is only being investigated by a select few stakeholders in the pharmaceutical industry. The aforementioned scenario is attributed to the surrogate licensing model, which has granted exclusive control of the associated intellectual property (IP) to three leading players, namely Editas Medicine, CRISPR Therapeutics and Intellia Therapeutics, in the contemporary market.

Clinical trials of CRISPR based therapeutics are currently focused mainly on oncological and hematological disorders; however, several product candidates against certain neurological disorders and infectious diseases, specifically targeting recurrent conditions, are under investigation. Post 2014, the overall interest in this technology has grown exponentially, with several start-ups entering the market and 6 of the top 10 pharmaceutical companies restructuring their efforts in this direction. Over time, a substantial body of evidence has also been generated validating the therapeutic applications of this technology, which has, in turn, prompted the establishment of numerous strategic partnerships (focused on therapy development and clinical research) and has caused investors to put in significant capital into innovator companies involved in this domain, over the last two years alone. In fact, the three leading companies in this industry segment together have combined market capitalization of more than USD 10 billion, and have raised more than USD 2.8 billion in various funding rounds. Despite the possibly limitless potential of the CRISPR/Cas technology, further investigation, probing its safety and therapeutic efficacy in large diverse populations, is required. Key impediments to approval and other existing challenges that are being addressed by stakeholders, include off-target toxicity-related concerns and complexities related to the delivery of CRISPR components into target cells. Concerning delivery, innovators in this field have reported notable success using different types of platforms for facilitating the intracellular administration of CRISPR components; examples of successful delivery methods include electroporation, AAV vectors and lipid nanoparticles (LNPs). A few companies are also evaluating bacteriophages as a potential delivery system for such products. Promising clinical results, and ongoing technical developments, coupled to the growing interest of biopharmaceutical developers, are anticipated to push pipeline products to higher phases and on to commercialization. We believe that the market is likely to evolve at a commendable pace over the next decade.

SCOPE OF THE REPORT The CRISPR Based Therapeutics Market, 2021-2030 report features an extensive study of the current market landscape and future opportunity for the players involved in the development of CRISPR based therapeutics for the treatment of a variety of disease conditions. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographies. Amongst other elements, the report includes: A review of the CRISPR based therapeutics that are currently in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as target therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others), phase of development (discovery, preclinical and clinical), approach of therapy (ex vivo and in vivo), cell source (autologous and allogeneic), type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), and the type of technology used. It also includes information on the completed, ongoing and planned clinical trials for CRISPR based therapeutics, sponsored by various industry players. Elaborate profiles of key players in this domain. Each company profile features a brief overview of the company, its financial information (if available), a brief description of its therapeutic candidates, recent developments, and an informed future outlook. An in-depth analysis of around 2,000 patents related to CRISPR technology that have been filed / granted, since 2015, highlighting the key trends associated with these patents, across type of patent, publication year and application year, regional applicability, IPCR symbols, emerging focus areas, inventor information, leading patent assignees (in terms of number of patents filed / granted), patent benchmarking and valuation. An analysis of the partnerships that have been inked by various stakeholders engaged in the development of CRISPR based therapeutics, during the period 2014-2020, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. An analysis of the investments made at various stages of development of the companies engaged in this field, covering instances of seed financing, venture capital financing, grants / awards, capital raised from IPOs and subsequent offerings. An analysis of the start-ups (established in the time period between 2013-2020 and have less than 200 employees) engaged in the development of CRISPR based therapeutics, based on several parameters, such as number of candidates in discovery, preclinical and clinical phase of development, therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

One of the key objectives of the report was to estimate the future growth potential of CRISPR based therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2021-2030. For this purpose, we have segmented the future opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL), [D] key geographical regions (North America, Europe and Asia-Pacific). To account for uncertainties and to add robustness to our model, we have provided three market forecast scenarios, portraying the conservative, base and optimistic tracks of the anticipated industrys growth.

KEY QUESTIONS ANSWERED Who are the leading players engaged in the development of CRISPR based therapeutics? Which key clinical conditions can be treated by CRISPR based drugs? What are the investment trends in this industry? Which partnership models are commonly adopted by stakeholders engaged in this domain? How has the intellectual property landscape in this market evolved over the years? Which factors are likely to influence the evolution of this market? How is the current and future market opportunity likely to be distributed across key market segments?

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been validated from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views

While the focus has been on forecasting the market till 2030, the report also provides our independent views on various non-commercial trends emerging in this industry. This opinion is solely based on our knowledge, research and understanding of the relevant market trends gathered from various secondary and primary sources of information.

CHAPTER OUTLINES Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the CRISPR therapeutics market and its likely evolution in the short-mid to long term.

Chapter 3 provides a general introduction to CRISPR/Cas system. In addition, we have briefly described the components of CRISPR/Cas system, its mechanism of action and vehicles to deliver CRISPR/Cas components in to the target cells. The chapter lays emphasis on the applications of CRISPR technology. It also includes a discussion on the challenges associated with the use of CRISPR based therapeutics.

Chapter 4 presents a detailed assessment of the current market landscape of CRISPR based therapeutics, along with information on type of therapy (CAR-T therapy, HSC therapy, T cell therapy, Phage therapy and others), approach of therapy (in vivo and ex vivo), cell source (autologous and allogeneic), phase of development (discovery, preclinical and clinical), type of delivery vehicle used (viral vector, electroporation, LNPs, bacteriophage and others), target disease indication and therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscular diseases, neurological disorders, oncological disorders, ophthalmic diseases and others). In addition, it provides an overview of the CRISPR based therapeutics developer landscape, highlighting the players that are active in this domain. It includes information on their year of establishment, company size (in terms of number of employees) and location of headquarters of the drug developers. We have presented a logo landscape, highlighting the distribution of the drug developers based on company size and location of headquarters. Further, it presents an analysis on the initiatives of the big pharma companies in this domain.

Chapter 5 includes profiles of the key players engaged in the development of CRISPR based therapeutics (shortlisted based on strength of product portfolio). Each profile features a brief overview of the company, its financial information (if available), brief details of gene editing technology, therapeutic pipeline, recent developments and an informed future outlook.

Chapter 6 provides an in-depth analysis of the patents filed / granted for CRISPR technology since 2015. The analysis also highlights the key trends associated with these patents, including type of patent (granted patent, patent application and others), publication year, application year, geographical location / patent jurisdiction (North America, Europe, Asia-Pacific and Rest of the World), IPCR symbols, key inventors and leading industry / non-industry players. In addition, it includes a detailed patent benchmarking analysis of leading players and patent valuation analysis, which evaluates the qualitative and quantitative aspects of these patents.

Chapter 7 features a detailed analysis of the partnerships and collaborations that have been inked in this domain since 2014, covering research and licensing agreements, R&D agreements, licensing agreements, licensing and manufacturing agreement, product development and manufacturing agreements, joint ventures and other types of partnership deals. The chapter includes analysis based on year of partnership, type of partnership model, purpose of licensing deal, and most active player(s) (in terms of number of partnerships inked). In addition, the chapter features a discussion on the surrogate licensing practice in the CRISPR based therapeutics market.

Chapter 8 provides an analysis of the investments made since 2014 at various stages of development of companies engaged in this domain, based on the year of investment, number of funding instances, amount invested and type of funding, highlighting most active players (in terms of number of funding instances and amount raised) and most active investors (in terms of number of funding instances).

Chapter 9 presents an analysis of the start-ups (established after 2012 and having less than 200 employees) engaged in the development of CRISPR based therapeutics based on the parameters, such as number of candidates in discovery, preclinical and clinical phase of development, target therapeutic area, amount raised through funding, number of investors, type of funding, number of deals signed, and number of patents filed.

Chapter 10 features an elaborate discussion on the future market potential of various CRISPR based therapeutics. The chapter provides insights on the likely distribution of the current and forecasted opportunity across [A] target therapeutic area (hematological disorders, oncological disorders, ophthalmic diseases, infectious diseases and others) [B] approach of therapy (ex vivo and in vivo), [C] type of therapy (CAR-T cell therapy, HSC therapy, T cell therapy, and TIL) and [D] key geographical regions (North America, Europe and Asia-Pacific).

Chapter 11 is a summary of the overall report, highlighting the key facts and figures related to the research and analysis presented in the previous chapters.

Chapter 12 is a collection of interview transcripts of discussions held with representatives of renowned organizations engaged in the CRISPR technology domain. In this chapter, we have presented the insights on our conversation with Harrison Wong (Public Relations, Burns McClellan, for eGenesis).

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix, which consists the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06130494/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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CRISPR Based Therapeutics Market by Type of Therapy, Therapeutic Approach, Therapeutic Area, and Key Geographical Regions : Industry Trends and Global...

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This article was originally published here

Life Sci. 2021 Aug 25:119908. doi: 10.1016/j.lfs.2021.119908. Online ahead of print.

ABSTRACT

Genetic disorders and congenital abnormalities are present in 2-5% of births all over the world and can cause up to 50% of all early childhood deaths. The establishment of sophisticated and controlled techniques for customizing DNA manipulation is significant for the therapeutic role in such disorders and further research on them. One such technique is CRISPR that is significant towards optimizing genome editing and therapies, metabolic fluxes as well as artificial genetic systems. CRISPR-Cas9 is a molecular appliance that is applied in the areas of genetic and protein engineering. The CRISPR-CAS system is an integral element of prokaryotic adaptive immunity that allows prokaryotic cells to identify and kill any foreign DNA. The Gene editing property of CRISPR finds various applications like diagnostics and therapeutics in cancer, neurodegenerative disorders, genetic diseases, blindness, etc. This review discusses applications of CRISPR as a therapeutic in various disorders including several genetic diseases (including sickle cell anemia, blindness, thalassemia, cystic fibrosis, hereditary tyrosinemia type I, duchenne muscular dystrophy, mitochondrial disorders), Cancer, Huntingtons disease and viral infections (like HIV, COVID, etc.) along with the prospects concerning them. CRISPR-based therapy is also being researched and defined for COVID-19. The related mechanism of CRISPR has been discussed alongside highlighting challenges involved in therapeutic applications of CRISPR.

PMID:34453943 | DOI:10.1016/j.lfs.2021.119908

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Applications of CRISPR as a potential therapeutic - DocWire News

Recommendation and review posted by Bethany Smith

CRISPR Clinopathomics is an emerging science with applications in biotechnology and cellular biology. It is a genome-wide, single-gene-based technology to which various pharmacological agents can be added to promote disease resistance. It has great potential to address all life sciences challenges with an approach that allows for the production of multiple proteins that can attack many of lifes common challenges. This technology can potentially address most life-threatening diseases such as cancer, infections, inflammation, inherited disorders, diabetes, aging, neurological and other disorders, and chronic conditions, and possibly more.

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*The Sample only consist ofTable of Content (ToC).Research Frameworkof the actual report.Research Methodologyadopted for it.

Major Company Profiles Covered in This Report:Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Technological advancements in the field of genome editing are expected to drive growth of the global CRISPR and CAS gene market during the forecast period. Key biopharmaceutical companies across the globe are focused on research and development activities, in order to innovate novel technologies in genome editing. For instance, in September 2020, Intellia Therapeutics Inc. presented new data demonstrating the efforts of In Vivo CRISPR/CAS9 edits to reduce a disease-causing protein or restore a functional protein.

Furthermore, in December 2020, Editas Medicine Inc., a genome editing company, submitted an investigational New Drug (IND) to the U.S. Food and Drug Administration (FDA), indicated for sickle cell disease. Such advancements and R&D activities have increased the demand for genome editing. Hence, these factors are expected to drive growth of the global CRISPR and CAS gene market during the forecast period. Moreover, increasing research in plant genome editing programs is expected to boost the global CRISPR and CAS gene market growth over the forecast period.

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However, off-target effects associated with CRISPR technology such as deletions, unintended point mutations, and translocations are expected to hamper the global CRISPR and CAS gene market growth over the forecast period. Among regions, North America is expected to witness significant growth in the global CRISPR and CAS gene market during the forecast period. This is owing to various companies involved in gene editing and the development of novel therapeutics across the region. Moreover, Asia Pacific is expected to register a robust growth rate over the forecast period, owing to increasing popularity of CRISPR technology in the region.

Key companies involved in the global CRISPR and CAS gene market are CRISPR Therapeutics, Inscripta, Inc., AstraZeneca, Mammoth Biosciences, Addgene, Synthego, Caribou Biosciences, Inc., Takara Bio, Inc., Cellectis, New England BioLabs, Editas Medicine, Inc., Merck KGaA, F. Hoffmann-La Roche Ltd., Intellia Therapeutics, Inc., and Danaher Corporation.

For instance, in November 2019, Caribou Biosciences Inc., a CRISPR genome editing company, announced the results of a new study demonstrating human genome engineering with TYPE I CRISPR-CAS systems.

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Shape Therapeutics CEO Francois Vigneault. (Shape Photo)

Shape Therapeutics has signed a deal potentially exceeding $3 billion with pharma giant Roche to support the development of gene therapies for Alzheimers and Parkinsons disease, the Seattle company announced Wednesday.

Shapes RNA editing technologies can change the sequence of RNA, which encodes the bodys protein building blocks. The company will deploy this technology with Roche in preclinical studies against targets relevant for these neurological conditions as well as certain rare diseases.

The collaboration may also leverage the biotech companys technologies for gene delivery. This system is designed to deliver RNA editing technology or other payloads directly to specific areas of the body, such as the nervous system or muscle.

Shape aims to unlock the next breakthrough in RNA technologies in the gene therapy space across a wide range of therapeutic areas, said Francois Vigneault, co-founder and CEO, in a statement.

Shapes RNA editing technology could potentially be used to change the amount of a key regulatory protein in the body or treat genetic diseases. The companys gene delivery technology is based on AAV vectors, a platform currently used to treat several rare conditions.

Under the agreement, Shape is eligible to receive an initial payment as well payments for hitting development, regulatory and sales milestones potentially exceeding $3 billion in aggregate value. Any products from the collaboration will be developed and commercialized by Roche.

The collaboration comes on the heels of a recent $112 million Series B investmentin June for the biotech, founded in 2018 with RNA editing technology spun out of the lab of co-founderPrashant Mali, a bioengineer at the University of California, San Diego.

Vigneault was former VP of research at Juno Therapeutics, a Seattle cell therapy company acquired by Celgene in 2018 for $9 billion. Other Juno veterans include Shapes head of platform technologies Adrian Briggs and vice president and head of research David Huss.

The new collaboration may reflect growing drug company interest in neurosciences, which is on the upswing after a lull several years ago. The recent FDA approval of Biogens Alzheimers drug Aduhelm may also be propelling interest in the field.

This new collaboration is also perfectly aligned with our broader efforts across the Roche Group to unlock the full potential of gene therapy, said James Sabry, Head of Roche Pharma Partnering, in a statement.

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Seattle biotech firm Shape Therapeutics inks gene therapy deal with Roche worth up to $3B - GeekWire

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Parkinsons disease is the second most common neurodegenerative disorder in the United States, affecting at least 500,000 people. Its a progressive disease, but can be managed with treatment. For now, the condition is diagnosed by symptoms rather than specific lab tests.

By the time a formal diagnosis is made, the National Institute of Neurological Disorders and Stroke (NINDS) notes that the disease has usually progressed to a point where people have difficulty controlling bodily movements.

Thats why its important to know whether its possible to prevent this disease.

Currently, there is no therapy or treatment that can slow the progression of Parkinsons or effectively relieve advanced symptoms, according to the NINDS.

By the time classic motor symptoms of Parkinsons disease show up, a significant loss of brain cells and function have already occurred. Scientists are investigating ways to detect early signs of the disease, to potentially stop or slow the progression.

Researchers aim to learn more about biomarkers of the early stages of the disease. Finding reliable biological identifiers might help doctors diagnose and treat Parkinsons earlier. Identifying these signs would give them more time to try therapeutic treatments before the disease has progressed.

For example, research indicates that it may be useful to study the activity of a neuronal protein in the brain known as -synuclein, or alpha-synyclein.

A 2019 study noted that measurements of -synuclein have shown encouraging preliminary results with regard to potential early diagnosis. Another 2019 study also examined how -synuclein accumulates in the brains of people suspected of having Parkinsons disease.This information could be used to develop therapies, perhaps antibody therapy, to prevent that accumulation from happening.

Scientists are also working to learn more about environmental factors and genetic factors that might contribute to the risk of developing Parkinsons. One recent genetic research breakthrough is the development of a DNA chip called NeuroX, which could potentially determine a persons risk, but more research is needed.

Parkinsons disease is the result of complicated combination of interconnected events, as one 2016 study described it. Since aging is the most common risk factor, future treatments may need to take degeneration of certain neurons into account.

While its not yet known if there are surefire ways to prevent Parkinsons disease, there are a few things experts recommend.

For example, you might try incorporating physical activity into your routine and eating a healthy and balanced diet for a variety of health reasons. So far, research into nutritional supplements is lacking. However, if you have specific dietary needs, talk to your doctor to see if supplementation is appropriate.

Could CBD oil help? Its possible, but we dont know for sure yet. Some research, including a 2018 review of studies, suggest that cannabidiol (CBD) might help prevent Parkinsons disease. However, the studies are mostly animal studies and there is not yet a body of research involving humans and CBD.

Some experts suggest that you may be able to delay some of the effects of Parkinsons disease through regular physical activity. Ideally this would include a combination of exercise that includes:

The NINDS has funded a number of studies to learn more about the impact of exercise, including whether exercise might help people delay the need for medication.

There are a variety of options for treating and managing Parkinsons symptoms, most of which involve medications that address the brains low levels of dopamine. Dopamine is a chemical in your brain that affects movement, and Parkinsons causes your brain to lose neurons that produce this chemical.

Medications that address this include levodopa, or levodopa combined with carbidopa. Or your doctor might prescribe a dopamine agonist, which mimics the action of dopamine in your brain. Other drugs used to treat Parkinsons include:

Another possible treatment option is deep brain stimulation (DBS). DBS was approved by the U.S. Food and Drug Administration in 1997. Many people have found that this treatment, which involves sending electrical impulses into the brain via tiny electrodes, helps control tremors once treatment with levodopa is no longer effective.

A small 2018 study found that DBS seemed to slow the progression of tremors in people with Parkinsons disease. It also found that DBS could be used effectively in people with an earlier disease stage than previously thought.

Scientists hope that more treatments may become available in the future, as they learn more about which drug may or may not be effective at slowing or halting the progression of the disease.

For example, a randomized, double-blind trial of 62 patients found that people with Parkinsons who took a drug usually used to treat diabetes seemed to stop the progression of the Parkinsons symptoms. They received weekly injections of exenatide for 48 weeks.

It was a relatively small study, and longer-term trials are needed, according to the researchers. A larger study involving more patients is currently ongoing.

If you are already living with Parkinsons disease, here are some tips to manage it:

There are medications that can help treat the symptoms of Parkinsons disease, and scientists are currently conducting research that could result in new treatment and therapies.

For example, you might one day have the option to take a medication used to treat prostate gland enlargement if youre at risk for developing Parkinsons disease.

The results of a study published in early 2021 suggest that certain medications often used to treat enlarged prostates are associated with a decreased risk of developing Parkinsons disease. Specifically, the researchers compared terazosin, doxazosin, and alfuzosin, which enhance energy metabolism, to tamsulosin, which is also used to treat benign enlarged prostates. They found that the latter did not seem to have the same effect.

The findings built on their previous research, which suggested that the use of terazosin, doxazosin, and alfuzosin was associated with slower progression and fewer complications in people with Parkinsons disease.

Researchers are also looking into the potential of stem cells to create new neurons to produce dopamine. They are also researching a protein called glial cell-derived neurotrophic factor, or GDNF, to potentially slow the progression of Parkinsons.

Ongoing research into a gene called LRRK2 or LARK2 and how it may interact with other genes related to Parkinsons disease is also promising, as it may shed light on how the disease progresses and how it might be halted.

For now, the symptoms of Parkinsons disease can be managed with medication and potentially deep brain stimulation. But research is underway to look for earlier methods of detection, as well as better treatments. Eventually, we might even have a way to prevent it from developing in the first place.

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How to Prevent Parkinson's Disease: Tips, Medications, and Research - Healthline

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We sat down for an interview with Professor Botond Roska, MD PhD, one of the worlds leading experts in the study of vision and the retina and last years recipient of the Krber Prize, to discuss the ground-breaking therapy he and his team have developed and already successfully used to return some vision to a handful of blind patients.

Can you tell us about the exact nature of your research for which you received the Krber Award?

It is sometimes difficult to know exactly what you get an award for, but I think I received it for two distinct but related scientific pursuits: firstly, my research on understanding vision, and secondly, for developing a treatment called optogenetic vision restoration therapy, through which a completely blind person can be sensitized to light, allowing them to see again.

FactProfessor Botond Roska, MD PhD, is a biomedical researcher and one of the foremost experts on vision, the retina, and treating diseases that cause blindness. He is a founding director of the Institute for Molecular and Clinical Ophthalmology Basel (IOB) in Switzerland and a Professor at the University of Basel. In 2019, he was awarded the Order of Saint Stephen, the highest national honor bestowed by Hungary, as well as the Louis-Jeantet Prize for Medicine. In 2020, he won the Krber European Science Prize for his research on a gene therapy that could be utilized to restore some vision to the fully blind, which he successfully implemented with his team for the first time this May.

What exactly does this procedure you developed entail?

It is a so-called two-component therapy. We inject a gene therapy vector into the eye, which is a small, virus-like particle with a DNA that encodes a light-sensitive protein. We target this protein at some elements of the blind retina. The vector also has a goggle that records the world, and then projects a picture onto the retina in a very specific way, in a particular color.

How effective is it now, and how effective can it potentially become?

The therapy restores a certain amount of vision, but not full sight. Based on the few patients we have had so far, the first of whom we recently published a paper about, the therapy allows patients to recognize objects, but it does not enable them to read, for instance.

However, there is a lot of room to improve. This is only the very first step, the very first optogenetic therapy.

What kinds of blindness or visual impairment can it help or cure?

It is useful in cases when someone is fully blind as a result of photoreceptor dysfunction, but their retina is still connected to their brain via an intact optic nerve.

How profound an effect do you think being able to heal peoples vision at a large scale might have on society?

Blindness is one of the conditions that people name as the worst affliction to have. In fact, in a recent survey conducted in the U.S., participants named it as the worst condition they could suffer from out of a whole host of common health problems, ranking it above cancer, Alzheimers, and other truly devastating diseases.

Our whole life is spent looking at phones and computers. Particularly during the pandemic, we could have almost no social interactions or opportunities to work without these, so that blind people were essentially cut off from the world apart from what little interaction they could have with it through hearing.

Therefore, we hope that such therapies, once they become widely available since they are in the clinical trial phase at the moment will improve many peoples lives tremendously.

Where do you think your research might lead you in the future?

There are three main directions. The first one is simple; as we discussed, we need to keep researching and improving optogenetic vision therapies, expanding them to target other cell types in the retina.

The second one is to do with the fact that if the optic nerve is missing, we cannot provide any therapies at the moment. This is something we are working on at my institute together with researcher Dniel Hillier, who is leading these efforts. We aim to find ways of restoring vision when there is no optic nerve.

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Hungarian-born researcher Dr. Botond Roska has been awarded the 2019 Louis-Jeantet Prize for Medicine for the discovery of basic principles of visual information processing and the development of therapeutic strategies, such as gene therapy, to restore vision in retinal disorders. Within the last few months, the neurobiologist (who celebrates his 50th birthday this year) received []Continue reading

The third is that most visual impairment is partial blindness, in which the method we developed cannot be used, so we are interested in looking into very large diseases affecting a lot more people. We want to try to slow down the degeneration or restore more vision to those with partial sight loss.

Why did you choose to dedicate your career to understanding human vision at the hardware level?

It was a chain of random events. I did not plan to be a vision researcher. I just wanted to understand things. When I finished my medical school, I was quite sure I did not want to treat patients for a living, I was more interested in understanding the human body. I decided to go into research, and I met someone who was researching the retina. I started my research in this field, and came up against an increasing number of questions that I found fascinating.

I went from topic to topic, physiology, virology, the molecular biology of the eye, then towards the end of my graduate studies, I read a paper that said it was possible to make cells light-sensitive using molecules form other organisms. At that time, I understood the retina quite well, and thought that I could combine my knowledge with these findings to try to design a therapy. My lab and myself are both interested in gaining scientific insight as well as designing therapies, and that is the path I plan to continue on for the rest of my career.

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The findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible, Botond Roska said.Continue reading

What is the worst and best part of the publicity youve received as a result of your success?

The best part is certainly that blind people can become informed that we are working on a therapy, which can provide them with some hope. It is also important for my institute, which can more easily recruit brilliant scientific minds who will come up with even better therapies down the line.

On the other hand, while there is nothing really bad about publicity per se, it can sometimes be time-consuming. Our paper about our first patient came out in May, and it has been downloaded around 90,000 times, with just about every country in the world reporting on our findings. And of course, all of the press outlets in all these countries bombarded our inboxes with requests. We were basically paralyzed for almost a month.

As someone who is called upon with increasing frequency to explain your cutting edge research, how do you approach the issue of science communication?

Communication is very important. It is part of our lives. In Switzerland specifically, it is taken very seriously, and it is part of our jobs as researchers to explain things to the public. We also have professionals to help us navigate the world of public relations.

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President Jnos der decorated neurobiologist Botond Roska with the Hungarian Saint Stephen Order on Tuesday, Hungarys national holiday. Hungarian-born Researcher Awarded for Helping People Regain Their Sight Each year on the holiday of the founder of our state we celebrate those Hungarians whose outstanding talent and achievements serve the well-being and prosperity of many others, []Continue reading

The difficulty today is finding the balance between our work and its communication to the world. Because our research reaches a tremendous number of people extremely quickly thanks to the global nature of social media and other platforms, we get a lot of requests for interviews and articles. The difficulty for us is balancing our responsibility to inform the public of our progress with actually making some. Sometimes, it feels very much like we are in the eye of the storm.

Many people consider you a likely future Nobel laureate. Do you believe you might receive the award?

I dont think about any of that. It does not occupy any of my time or of my imagination. At this moment, there are still great challenges ahead of us if we want to make the therapies as good as they can be. That is what I concentrate on. We also need to focus on innovating and coming up with new tools to help realize our scientific vision. This is also a costly and time-consuming endeavor.

What would you tell aspiring scientists, how can they best succeed in their scientific endeavors?

I think that the key is to come to science with an incredible desire to solve interesting problems. I never wanted to be successful in the public eye, just accomplished at solving scientific questions. For some reason, some scientists are more present in the media, and some of them win prizes, while others win prizes and choose not to have a public presence. Often, the most brilliant and prolific scientists do not even win prizes.

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Returning Vision to The Fully Blind - Interview with Dr. Botond Roska - Hungary Today

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Ryder Comer was born with SMA, the best treatment option was an infusion that cost $2.1 million. Now, his mom is working to add SMA to Idaho's newborn screening.

MIDDLETON, Idaho When Jake and Haley Comer of Middleton welcomed their new baby boy Ryder into the world, they were completely overjoyed. But, they quickly realized something might not be right.

"The whole pregnancy was completely healthy, there were no signs of anything." said Haley Comer, Ryder's proud mom. "As soon as he was born though, he was diagnosed with low muscle tone. No parent wants to think that something is wrong with their perfect little baby."

The Comers went to their pediatrician to get some answers. The doctor agreed that newborn Ryder was "floppy" and had low muscle tone. He recommended genetic testing to see if there was a reason for that. While waiting for the results in the following weeks, the unthinkable happened. Ryder stopped breathing.

"The day after Ryder turned a month old, I found him unresponsive at home, I had him in a little baby carrier. So, I called 911 and started CPR and luckily was able to revive him. We were rushed to the ER, and that initiated a month-long stay in the hospital," said Haley Comer with tears in her eyes.

While they were at St. Luke's, the genetic testing results came back. The news was not what the Comers were hoping for.

"We got our test results back and Ryder was diagnosed with SMA, Spinal Muscular Atrophy Type 1," said Haley. "It was hard, there were a lot of tears, but at the same time we want to be brave. You have to put on a brave face for your baby."

SMA is a progressive, rare genetic disease that impacts the muscles. Babies with SMA Type 1 face many physical challenges, including muscle weakness and trouble breathing, coughing, and swallowing.

Doctors at St. Luke's in Boise told the Comers about a promising new treatment option. A gene therapy infusion that could save baby Ryder's life.

"They told us that they would be transferring him to Salt Lake City to Primary Children's Hospital, because no hospitals in Idaho performed this infusion," said Haley. "A couple days later, we met with a neurologist in Salt Lake and learned about our treatment options. This gene therapy option seemed like the best one. It's a once in his lifetime, one-hour long, gene therapy infusion. He is missing a very important gene that produces survival motor neuron protein. That helps our body move our muscles. It basically injects this gene into his body, and then his body learns to keep producing this survival neuron protein to keep his muscles alive."

#7sHero Tonight at 10 p.m. on KTVB youll meet this precious baby boy, Ryder Comer of Middleton. His parents Jake &...

It sounded like the best option for Ryder, but the Comers were stunned when they found out just how much the infusion treatment would cost.

"It's called Zolgensma, and it costs $2.1 million for one single infusion," said Haley.

Zolgensma is FDA-approved for patients with all forms and types of SMA who are under 2 years of age. Research shows babies dosed with Zolgensma as soon after diagnosis as possible have had better results than those who waited to begin treatment. It's given through an IV infusion that takes about an hour, and it's a one-time treatment.

The Comers weren't even sure their insurance would cover it, but they moved forward with trying to get the pricey infusion procedure preauthorized.

"It was terrifying when they told us," said Jake Comer, Ryder's dad. "I was like I hope you take monthly payments for the rest of my life."

Incredibly, the approval from their insurance company, United Healthcare, went smoothly and quickly.

"We paid our deductible, and our out-of-pocket, and they took care of the rest," said Jake in disbelief. "That definitely took the stress of our shoulders during such a traumatic time, so we are very grateful."

The Comers say it was all a whirlwind. Ryder received the infusion within 24 hours.

"We feel pretty fortunate because a lot of families had to fight for weeks and months to get their insurance to cover this," said Haley. "For Ryder, his disease had progressed so much by the time he got it, he was at the point where every day mattered."

After the $2.1 million infusion, Ryder started making strides within days. The changes they've seen over the past several months have been so wonderful for the Comers.

"His breathing got stronger, his voice got stronger, his cry got louder, he had a really weak cry at first," said Haley. "Now he can lift his arms above his head and grab toys! He can't quite hold his head up yet, but we are working on it. He is in feeding and physical therapy, and we are doing everything we can to get him stronger."

So far, Ryder is the only baby in Idaho with SMA to receive this gene therapy infusion. Haley has a new passion. She is now dedicated to helping other SMA families like theirs.

"What we are really working toward right now is to have SMA added to Idaho's newborn screening. Idaho is one of the last remaining states that does not include SMA on its newborn screening test. It's just a simple blood test. Before the approval of his gene therapy two years ago, the life expectancy for babies diagnosed with SMA Type 1 was not much past two years old. That is just devastating for families. So having SMA added to Idaho's newborn screening will allow these babies to have access to treatment so much sooner. The sooner you get a diagnosis and have treatment, the better outcomes you will have in life."

Haley has been in contact with the Idaho Department of Health and Welfare, and with the organization CURE SMA, which has been working to add this disease to every state's newborn screening. She's in the early stages, but this mom's goal is to make that happen here in Idaho, too.

"We just think that every baby deserves to be treated as soon as they possibly can," said Haley.

August is SMA Awareness Month. Baby Ryder and his parents are paving the way for other Idaho babies born with SMA.

"He's expected to get stronger and hopefully meet those milestones of walking and talking and crawling and all those things, but we just don't know, we are working hard in therapy to give him the best possible chance. One day a time."

For more information on SMA, click here. If you'd like to help the Comer family with their medical expenses, there is a GO FUND ME set up to do so.

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Ryder's Story: Middleton baby with rare genetic condition gets a $2.1 million infusion to give him a fighting chance - KTVB.com

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NEW YORK An international team of researchers has used transcriptomic data from ulcerative colitispatients to develop a predicted polygenic transcriptional risk score, or PPTRS,that can identify UC-affected individuals at fivefold elevated risk of progressing to surgical resection of the large bowel.

In a paper published on Thursday in the American Journal of Human Genetics, the Georgia Institute of Technology-led team noted that 5 percent to 10 percent of people with UC require bowel resection, or colectomy, within five years of diagnosis, but that polygenic risk scores based on genome-wide association studies generally don't provide meaningful prediction of progression to surgery. However, studies of Crohn's disease have shown that gene expression profiling of GWAS-significant genes provides some stratification of risk of progression to complicated disease through transcriptional risk scoring, or TRS.

In their paper, the researchers demonstrated that a measured TRS based on bulk rectal gene expression in a cohort of UC patients had a positive predictive value approaching 50 percent for colectomy. Single-cell profiling demonstrated that the disease-associated genes were active in multiple diverse cell types from both the epithelial and immune compartments, and expression quantitative trait locusanalysis identified genes with differential effects at baseline and the one-year follow-up, the researchers said. But for the most part, they found that differential expression associated with colectomy risk was independent of local genetic regulation.

Overall, their data suggested that prediction of gene expression from relatively small transcriptome datasets can be used in conjunction with transcriptome-wide association studies for stratification of risk of disease complications.

The researchers began by performing differential expression analysis between baseline rectal RNA-seq biopsies of individuals in the PROTECT multicenter pediatric inception cohort study of response to standardized colitis therapy. Analyses were done on 21 affected individuals who progressed to colectomy and 310 who did not. They identified downregulation of 783 transcripts in the individuals who underwent colectomy and upregulation of 1,405 transcripts overall.

They also obtained rectal biopsy RNA-seq data for 92 affected individuals at week 52 and observed a marked shift in gene expression at follow-up, prompting them to ask whether local regulation of the gene expression might contribute to this effect. They found that there were 72 SNPs that were significantly regulating 308 genes at both time points.

Further examination of the expression of colectomy-associated genes in a single-cell RNA-seq dataset obtained from rectal biopsies provided strong evidence that both epithelial and immune cells contributed to the risk of disease progression, the researchers said.

The researchers then performed a TWAS to capture the effects of all polymorphisms within 1 Mb of each transcript expressed in the PROTECT rectal biopsies and then used the weights to predict gene expression in a validation cohort from the UK Biobank. They tested for differential predicted gene expression in 70 percent of the validation samples and discovered about 800 genes either upregulated or downregulated in UC-affected individuals relative to non-IBD control individuals. They then derived a PPTRS for UC based on the effect sizes of the minor alleles and applied it to the remaining 30 percent of the validation samples, as well as to the PROTECT genotypes, and found that the PPTRS efficiently discriminated UC-affected individuals from non-IBD control individuals.

Significantly, it also discriminated the individuals who underwent colectomy versus those who didn't in both the UK Biobank and PROTECT.

"More extensive single-cell profiling, combined with cell-type-specific genetic analysis of gene expression, is likely to lead to the development of even better transcriptional risk signatures," the authors concluded. "It is also likely that such focused and personalized analysis may highlight specific pathological mechanisms active in particular affected individuals."

They did note, however, that these results were limited by the relatively small sample size of colectomies in the PROTECT study, and that validation of cross-ancestry assessments and the evaluation of the consistency of gene expression prediction across populations should be a high priority.

In an email, corresponding author and GIT researcher Greg Gibson noted that while the study's multiple layers of replication show that transcriptional profiling of the rectum greatly enhances risk stratification for risk of colectomy, this was not a clinical trial, so the approach is not yet approved for evaluation of patients.

"We hope that it will progress to implementation in the near future," he added."The prediction from genotypes alone is less likely to have clinical utility since the precision is still quite low, so that aspect is more research oriented."

He further noted that the approach he and his colleagues used could also be applied to a wide range of diseases, and that they are pursuing that research.

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Ulcerative Colitis Study Analyzes Gene Expression to Measure Risk of Progression to Surgery - GenomeWeb

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Brain development that occurs after birth is also important. Rebecca Saxe at MIT is working to understand the brain structures and activities responsible for social cognition, which allows us to consider the mental states of other people.

Saxe has discovered a particular brain region that is key; by studying how activity in this region and others changes over the course of childhood, she may be able to understand how social abilities develop. She has also found that these brain activity patterns are altered in people with autism spectrum disorders.

Even though researchers are starting to understand some of the processes that govern development and have identified things that can derail it, were far from being able to intervene when such problems occur. But as we gain insights, we could someday test therapies or other ways to address these developmental issues.

Computational neuroscientists use mathematical models to better understand how networks of brain cells help us interpret what we see and hear, integrate new information, create and store memories, and make decisions.

Understanding how the activity of neurons governs cognition and behavior could lead to ways to improve memory or understand disease processes.

Terry Sejnowski, a computational neurobiologist at the Salk Institute, has built a computer model of the prefrontal cortex and analyzed its performance on a task in which a person (or machine) has to sort cards according to a rule thats always changing. While humans are great at adapting, machines generally struggle. But Sejnowskis computer, which imitates information flow patterns observed in the brain, performed well on this task. This research could help machines think more like humans and adapt more quickly to new conditions.

Aude Oliva, the MIT director of the MIT-IBM Watson AI Lab, uses computational tools to model and predict how brains perceive and remember visual information. Her research shows that different images result in certain patterns of activity both in the monkey cortex and in neural network models, and that these patterns predict how memorable a certain image will be.

Research like Sejnowskis may inspire smarter machines, but it could also help us understand disorders in which the function of the prefrontal cortex is altered, including schizophrenia, dementia, and the effects of head trauma.

Researchers are trying to determine the genetic and environmental risk factors for neurodegenerative diseases, as well as the diseases underlying mechanisms.

NHUNG LE

Improving prevention, early detection, and treatment for diseases like Alzheimers, Parkinsons, Huntingtons, chronic traumatic encephalopathy, and ALS would benefit millions of people around the world.

Yakeel Quiroz, at Massachusetts General Hospital, studies changes in brain structure and function that occur before the onset of Alzheimers symptoms. Shes looking for biomarkers that could be used for early detection of the disease and trying to pinpoint potential targets for therapeutics. One potential biomarker of early-onset Alzheimers that shes founda protein called NfLis elevated in the blood more than two decades before symptoms appear. Quiroz has also identified a woman with a protective genetic mutation that kept her from developing cognitive impairments and brain degeneration even though her brain showed high levels of amyloid, a protein implicated in Alzheimers development. Studying the effects of this beneficial mutation could lead to new therapies.

Researchers at the Early Detection of Neurodegenerative Diseases initiative in the United Kingdom are analyzing whether digital data collected by smartphones or wearables could give early warnings of disease before symptoms develop. One of the initiatives projectsa partnership with Boston Universitywill collect data using apps, activity tracking, and sleep tracking in people with and without dementia to identify possible digital signatures of disease.

As we learn more about the underlying causes of neurodegenerative diseases, researchers are trying to translate this knowledge into effective treatments. Advanced clinical trials targeting newly understood mechanisms of disease are currently under way for many neurodegenerative disorders, including Alzheimers, Parkinsons, and ALS.

Connectomics researchers map and analyze neuronal connections, creating a wiring diagram for the brain.

Understanding these connections will shed light on how the brain functions; many projects are exploring how macro-scale connections are altered during development, aging, or disease.

Mapping these connections isnt easythere may be as many as 100 trillion connections in the human brain, and theyre all tiny. Researchers need to find the best ways to label specific neurons and track the connections they make to other neurons in remote parts of the brain, refine the technology to collect these images, and figure out how to analyze the mountains of data that this process produces.

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Eight ways scientists are unwrapping the mysteries of the human brain - MIT Technology Review

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State College, Pennsylvania., August 30, 2021 / PRNewswire / -NeuExcell Therapeutics (www.neuexcell.com), A gene therapy company focused on neurodegenerative diseases has announced a Series Pre-A funding round of over $ 10 million. The round was led by Co-Win Ventures and was attended by other institutional investors Yuan Bio, Oriza Seed, Tsingyuan and Inno Angel.

We are honored to join this very reputable group of investors, he said. Peter Tombros, Chairman of the Board of Directors of NeuExcell Therapeutics. Investor experience and support will enable us to leverage our unique neuroregenerative gene therapy platform across multiple neurodegenerative indications. This funding strength validates our strategy and biotechnology. Further examine our science in the industry.

Professor Gong Chen, co-founder and chief scientific advisor of the company, said: There is an urgent need for breakthrough therapies like us.

I think this is a great opportunity to invest in experienced leadership, he said. Xin Huang, Managing Partner of Co-Win Ventures. NeuExcells unique technology has the potential to act as a platform for treating many neurodegenerative diseases, providing hope for breakthrough new therapies for patients who do not have the right choices today.

With the end of this successful pre-A round, we welcome him. Xin Huang Jonathan Sun attended the board meeting.

About NeuExcellTherapeutics

NeuExcell is a privately held early stage genetic engineering company headquartered in Pennsylvania, USA When Shanghai, China.. Its mission is to improve the lives of patients suffering from neurodegenerative diseases and damage to the central nervous system. Based on Professor Gong Chens scientific research, we have developed a potentially destructive nerve repair technique through the conversion of astrocytes to neurons. In vivo By introducing neural transcription factors through adeno-associated virus (AAV) -based gene therapy. NeuExcells pipeline covers major neurodegenerative diseases such as stroke, Huntingtons disease, amyotrophic lateral sclerosis (ALS), Alzheimers disease, Parkinsons disease, traumatic brain injury, spinal cord injury, and glioma. increase.

About Co-Wof Venture

Founded in 2009, Co-Win Ventures is an early stage investor in healthcare and TMT with a focus on equality, transparency, sharing and innovation. Co-Wins business network China When USA..Total AUM is about US $ 1 billion, Co-Win aims to be a reliable partner for great entrepreneurs to build breakthrough technologies and businesses. Co-Win Ventures has helped more than 140 portfolio companies, including leading leaders in their respective sub-sectors, including Cytek, Connect, Thrive (acquired by Nasdaq-listed company EXAS), Taimei Technology, Genecast, Sinovation and Augta. ..

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Source NeuExcellTherapeutics

NeuExcell Therapeutics Raises Over $ 10 Million Series Before Round To Keep The Company Growing | Around The Web-Pennsylvania

Source link NeuExcell Therapeutics Raises Over $ 10 Million Series Before Round To Keep The Company Growing | Around The Web-Pennsylvania

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NeuExcell Therapeutics Raises Over $ 10 Million Series Before Round To Keep The Company Growing | Around The Web-Pennsylvania - Pennsylvania News...

Recommendation and review posted by Bethany Smith

SAN FRANCISCO--(BUSINESS WIRE)--AllStripes (Company), a healthcare technology company dedicated to unlocking treatments for people affected by rare diseases, today announced the Company completed a $50 million Series B financing round.

The financing was led by Lux Capital, a current investor, joined by JAZZ Venture Partners, Spark Capital, Medidata Solutions (a Dassault Systmes company), McKesson Ventures, and Maveron, along with angel investors including Arif Nathoo, CEO of Komodo Health, and Leila Zegna, Director of the Kabuki Syndrome Foundation.

The funding will support launching 100 new rare disease research programs while expanding global operational footprint, technology and data automation enhancements to improve research insights, further developing the platform capabilities to enhance the user experience and strengthen the life sciences offerings, and continuing to invest in growing the companys team to support creating the playbook for rare disease research.

Beginning research on a rare condition can feel like being dropped into a new world without a map and we are on a mission to change that with data, said Nancy Yu, CEO and Co-founder, AllStripes. Were proud that so many of our current investors have recognized the progress weve made and are continuing to support our vision to transform rare disease research. This investment will allow us to better support the rare disease community, where each persons experience is essential to understanding disease progression - ultimately leading to new treatments for rare disease patients around the globe.

AllStripes has a successful track record of bringing together patient organizations, families, experts and life science partners to advance research, said Adam Goulburn, Partner, Lux Capital and AllStripes board member. If we are going to improve rare disease treatments, these groups must work together. We believe in AllStripes vision to transform rare disease research and empower patients. If the past year showed us anything, it is that patients want a voice when it comes to their health, and their engagement with research is invaluable.

There are more than 7,000 rare diseases and only 5 percent have treatments. By working with AllStripes, we hope to improve the number of treatments available by accelerating research for rare diseases, said James M. Wilson, M.D., Ph.D., Rose H. Weiss Professor and Director, Orphan Disease Center; Director, Gene Therapy Program at the University of Pennsylvania; Professor in Departments of Medicine and Pediatrics, Perelman School of Medicine.

AllStripes is currently partnering with more than 30 patient advocacy organizations across its 40 conditions, supporting more than 3,000 users to date. As a public benefit corporation (PBC), AllStripes is advocating for the importance of real-world evidence in development of treatments and is continuing to build tools that make research more inclusive for the global rare disease community. The Company is committed to transparent data collection and sharing data across the life sciences continuum to advance clinical research.

AllStripes collaborates with various biopharmaceutical companies and other entities on real-world evidence studies, including: HemoShear Therapeutics, Inc., Orphan Disease Center at the University of Pennsylvania, Taysha Gene Therapies, Inc., UCB Biopharma SRL, and Novartis Pharma AG. These joint research programs aim to enhance clinical understanding of rare disease treatment research. AllStripes and the University of Pennsylvania's Orphan Disease Center are focused on clinical understanding of Lesch-Nyhan disease and Crigler Najjar syndrome type 1, with the goal of facilitating therapeutic discovery for both conditions. One of AllStripes most recent partnerships with HemoShear Therapeutics aims to gather real-world evidence on patients' medical experience with serious metabolic diseases through the Journey to Understand MMA and PA (JUMP) study.

To learn more about AllStripes, its partners and its commitment to the rare disease community, please visit allstripes.com.

About AllStripesAllStripes is a healthcare technology company dedicated to unlocking new treatments for people with rare diseases. AllStripes has developed a technology platform that generates regulatory-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, JAZZ Venture Partners, Spark Capital, Medidata Solutions, McKesson Ventures, Maveron, and a number of angel investors. For more information, visit http://www.allstripes.com.

About Lux CapitalLux Capital invests in emerging science and technology ventures at the outermost edges of what is possible. We partner with iconoclastic inventors challenging the status quo and the laws of nature to bring their futuristic ideas to life. Over the past two decades, Lux has expanded from its New York City roots to Silicon Valley, and built a $4 billion AUM firm of more than 30 full-time professionals, with the versatility to invest at any stage.

JAZZ Venture PartnersJAZZ Venture Partners invests in companies that extend the boundaries of human performance improving how we live, learn, work, play, and experience the world. JAZZ seeks breakthroughs at the frontiers of technology and science, such as advances in artificial intelligence, neurobiology, augmented reality, and closed-loop human-computer systems. JAZZ portfolio companies are unlocking human potential in health, mind-body wellness, accelerated learning and training, sports, entertainment, and the enterprise. More information about JAZZ can be found at http://www.jazzvp.com.

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AllStripes Announces $50 Million Series B Financing to Advance Global Rare Disease Research - Business Wire

Recommendation and review posted by Bethany Smith

VANCOUVER, British Columbia & BASEL, Switzerland--(BUSINESS WIRE)--Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, today announced that it has entered into a global collaboration and license agreement with Takeda Pharmaceutical Company Limited (Takeda) for the development and commercialization of novel nonviral gene therapies to treat up to two undisclosed rare liver diseases. This is the second collaboration between Genevant and Takeda, following an earlier 2021 agreement to develop nucleic acid therapeutics directed to specified targets in hepatic stellate cells to treat liver fibrosis.

LNP provides a compelling approach to deliver on the promise of gene therapy, and our leadership position in the LNP space is well established. We have enjoyed working with our Takeda colleagues to develop hepatic stellate cell-directed treatments to treat liver fibrosis and are delighted to expand the relationship further with this second collaboration, said Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation.

Building on our existing foundation with Genevant in liver fibrosis, were excited to expand our work together to develop life-altering, nonviral gene therapies for specified rare liver diseases, said Bernard Allan, head of liver disease research at Takeda. Genevants expertise in the development of LNPs for clinical applications, coupled with Takedas drug development capabilities and history in gastroenterology, gives us a great opportunity to develop new treatment options for patients with liver disorders.

Under the terms of the agreement, Genevant is eligible to receive up to $303 million in upfront and potential milestone payments, plus royalties on future product sales. Takeda has exclusive rights to utilize Genevants LNP technology in the development and commercialization of specified nonviral gene therapies for up to two undisclosed rare liver diseases.

About Genevant Sciences

Genevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industrys most robust and expansive lipid nanoparticle (LNP) patent estate, and decades of experience and expertise in nucleic acid drug delivery and development. The Companys scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Companys LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. For more information, please visit http://www.genevant.com.

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Genevant Sciences Announces Global Collaboration and License Agreement with Takeda to Develop Novel Nonviral Gene Therapies for Up to Two Rare Liver...

Recommendation and review posted by Bethany Smith

Using cells from brain tumour patients as a 3D printing material, researchers have made a model of their tumour to test the efficacy of potential treatments before using them for real inside the body. "A chunk" of the tumour from a glioblastoma patient is used to print a model matching their MRI scans. The patient's blood is then pumped through the printed tumour, made with a compound that mimics the brain, followed by a drug or therapeutic treatment. While previous research has used such "bioprinting" to simulate cancer environments, the Tel Aviv University scientists involved in this research say they are the first to print a "viable" tumour.

Medulloblastoma, whilst being a common paediatric malignant central nervous system tumour, represent a small proportion of brain tumours in adults. This research reports on theunique subgroup-specific cytogenetic features of adult medulloblastoma, which are distinct from those in younger patients, and correlate with survival disparities. The findings suggest that clinical trials that incorporate new strategies tailored to high-risk adult medulloblastoma patients are urgently needed. These findings complement theimportant progress being made on medulloblastoma by researchers at our Research Centre at QMUL.

Chronobiology is a promising area for research in our area as circadian clock genes are linked to tumour biology and outcomes in multiple cancers, including glioma. In this research, the relationship between circadian clock genes, IDH mutational status, and prognosis in glioma patients was examined to try and gain further understanding into theassociation between circadian clock gene expression, the glioma microenvironment and patient survival.

Researchers in Brazil have discovered a set of biomarkers that can be used to predict which patients diagnosed with glioblastoma may have tumours that are more resistant to radiation therapy. This discovery could help doctors choose the treatment that should enable patients to live longer. Glioblastoma usually has to be surgically removed, followed by chemotherapy or radiation therapy, or both, to eliminate what could not be surgically removed and ensure the tumour does not return. However, patient survival tends to be low owing to the tumours resistance to treatment. According to most estimates, 80% of patients submitted to radiation therapy suffer a relapse a few months later. These researchers claim that their study provides important information for use in screening patients prior to deciding whether radiation therapy should be used. Its a large signature. We identified 31 genes that may indicate resistance or sensitivity to radiation when altered. So, its possible to verify whether there are tumour cells that are highly resistant to radiation, potentially assisting physicians in deciding on the optimal treatment.

Some facts and figures from the American Cancer Society in a new report illustrating thatalthough incidence rates of adult brain tumours are decreasing 5-year survival rates remain low.

There are over 120 different types of brain tumours and although distinguishing between them can be difficult it is nevertheless essential for effective treatment. This year, the Gertrud Reemtsma Foundation isawarding the International Prize for Translational Neuroscience to two researchers who have made a significant contribution to improving the diagnosis of brain tumours. Hai Yan from Duke University School of Medicine was instrumental in identifying two tumour proteins that are typical for certain sub-groups of gliomas. Andreas von Deimling and his group from the University of Heidelberg have developed antibodies that bind to these altered proteins. The tumours can now be classified at the molecular level. A patients cancer can thus be classified more precisely and the therapy adapted accordingly.

There wont be a research update next week but it will return on 10th September.

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3D printed tumours, adult medulloblastoma and the circadian clock - Brain Tumour Research

Recommendation and review posted by Bethany Smith

The latest Cell and Gene Therapy Market report serves as a valuable resource for data concerning the industrys trajectory over the forecast duration. It critically examines the key growth stimulants, bottlenecks, and rewarding prospects that are influencing the industry dynamics. Moreover, the literature consists of detailed segmentation data, as well as in-depth profiles of key companies in this domain.

Experts analysts state that Cell and Gene Therapy market size is anticipated to amass notable gains, accumulating a valuation of XX USD over the projected timeframe 2021-2026. In addition, projections for the various sub-markets are also validated in the report. Proceeding further, the research literature is well equipped with different methodologies to assist the stakeholders in seamlessly navigating any form of business crisis.

Key highlights from the Cell and Gene Therapy market report:

Request Sample Copy of this Report @ https://www.nwdiamondnotes.com/request-sample/3811

Cell and Gene Therapy market segments covered in the report:

Product gamut: Rare Diseases , Oncology , Hematology , Cardiovascular , Ophthalmology , Neurology and Other Therapeutic Classes

Application spectrum: Pharmaceutical and Biotechnology Companies , Research and Academic Institutions , Contract Research Organizations (CROs) , Hospital and Others

Competitive dashboard:

Regional bifurcation:

Key Questions Addressed by the Report

What Are the Key Opportunities in Global Cell and Gene Therapy Market?

What Will Be the Growth Rate from 2021 to 2026?

What Are the Factors That Will Impact/Drive the Market?

Which Segment/Region Will Have Highest Growth?

What is the Role of Key Players in the Value Chain?

What is the Competitive Landscape in the Industry?

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Cell and Gene Therapy Market Forecasts to 2026: Global Industry Growth, Share, Size, Trends and Segmentation Report - Northwest Diamond Notes

Recommendation and review posted by Bethany Smith

The latest published report namely Cancer Gene Therapy Market Growth 2021-2028 added by DBMR offers an insightful take on the historical data of the market and predictions for 2021 to 2028 time-period. The report provides an understanding of the Cancer Gene Therapy industry competitors, the sales channel, growth potential, potentially disruptive trends, industry product innovations and the value/volume of size, market segments, and market share of the best actors/products. Current market trends and dynamics are assessed which helps in mapping the track of the global market. A chapter-wise format has been used to ease the readability and complexity of the data. Each chapter is further categorized into its respective segments containing well-structured data.

According to DBMR, the GlobalCancer Gene Therapy Marketis expected to see a growth rate of 32.54%. The high success rate of cancer gene therapy along with clinical trial and the preclinical trial is gaining popularity among the patient which is leading towards the market.

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Market Overview of Global Cancer Gene Therapy

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period. High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

The topic of market segmentation covers research and analysis based on application, vertical, deployment model, end user, and geography. In addition, competitive analysis assists to get ideas about the strategies of key players in the market via an international Cancer Gene Therapy report. Some of these strategies can be named as; new product launches, expansions, agreements, partnerships, joint ventures, acquisitions, and others that help to broaden their footprints in the Cancer Gene Therapy industry. The market share of key competitors on global level is studied where main regions such as Europe, North America, Asia Pacific and South America are covered in the reliable Cancer Gene Therapy market report.

To Get the Short-Term and Long-Term Impact of COVID-19 on this Market@https://www.databridgemarketresearch.com/request-covid-19/global-cancer-gene-therapy-market

The titled segments and sub-section of the market are illuminated below

By Therapy (Oncolytic Virotherapy, Gene Induced Immunotherapy, Gene Transfer)

By End User (Hospitals, Oncology Institutes, Biotechnological Companies, Clinical Research Laboratories)

Top Key Manufactures or Players (this may not be a complete list and extra companies can be added upon request):

Adaptimmune

GlaxoSmithKline plc

bluebird bio, Inc

Merck & Co., Inc

CELGENE CORPORATION

Anchiano Therapeutics

Achieve Life Sciences

..

Complete Report is Available (Including Full TOC, List of Tables & Figures, Graphs, and Chart)@https://www.databridgemarketresearch.com/toc/?dbmr=global-cancer-gene-therapy-market

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Based on therapy, the cancer gene therapy market is segmented into oncolytic virotherapy, gene induced immunotherapy and gene transfer. The oncolytic virotherapy is further sub-segmented into adenovirus, lentivirus, retro virus, adeno associated virus, herpes simplex virus, alpha virus, vaccinia virus, simian virus and others. The gene induced immunotherapy is further sub-segmented into delivery of cytokines gene and delivery of tumor antigen gene. The gene transfer is further sub-segmented into naked/plasmid vectors, electroporation, sonoporation, magnetofection and gene gun.

Cancer gene therapy market has also been segmented based on the end user into hospitals, oncology institutes, biotechnological companies and clinical research laboratories.

According to the Regional Segmentation the Cancer Gene Therapy Market provides the Information covers following regions:

North America

South America

Asia & Pacific

Europe

MEA (Middle East and Africa)

The key countries in each region are taken into consideration as well, such as United States, Canada, Mexico, Brazil, Argentina, Colombia, Chile, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Belgium, Austria, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, China, Japan, Taiwan, South Korea, Singapore, India, Australia and New Zealand etc.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

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Scope of Report:

The Market report lists the most important competitors and provides the insights strategic industry Analysis of the key factors influencing the market. This report will help you to establish a landscape of industrial development and characteristics of the Cancer Gene Therapy market. The Global Cancer Gene Therapy market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, price, cost, revenue and gross margins.

Important Features that are under offering & key highlights of the report:

Market Data Segmentation with production, consumption, revenue (million USD), and Price Analysis

Detailed overview of Cancer Gene Therapy market

Changing market dynamics of the industry and Impact of Influencing Factors

In-depth market segmentation by Type, Application and other major segments etc.

To analyse and forecast the Global Cancer Gene Therapy market, in terms of value and volume.

Which segment has the potential to gain the highest market share?

To help decision maker from new offer perspective and benchmark existing marketing strategy.

Correlate cost structure historical data with key business segments.

Analyse marketing contribution and customer acquisition by up-selling and cross selling.

Identifying Influencing factors keeping Global Cancer Gene Therapy Market Intense, factored with periodic analysis of CR4 & CR8 concentration ratio & HHI Index.

Order a Copy of Cancer Gene Therapy Market Report@https://www.databridgemarketresearch.com/checkout/buy/singleuser/global-cancer-gene-therapy-market

With tables and figures helping analyze worldwide Global Cancer Gene Therapy market, this research provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

Major Highlights of TOC:

Chapter One: Market Overview

1.1. Introduction

1.2. Scope/Objective of the Study

Chapter Two: Executive Summary

2.1. Introduction

Chapter Three: Market Dynamics

3.1. Introduction

3.2. Market Drivers, Trends, Challenges, Opportunities

Chapter Four: Market Factor Analysis

4.1. Porters Five Forces

4.2. Supply/Value Chain

4.3. PESTEL analysis

4.4. Market Entropy

4.5. Impact Analysis Post COVID-19

Continued !!!

Chapter Nine: Methodology and Data Source

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

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Cancer Gene Therapy Market 2021 Global Industry Size, Share, Growth, COVID-19 Impact Analysis, and Drivers Analysis Research Report 2027|...

Recommendation and review posted by Bethany Smith

INTRODUCTION Given their ability to treat a wide range of clinical conditions, advanced therapy medicinal products, including cell and gene therapies, have revolutionized the delivery of healthcare.

New York, Aug. 24, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell Therapy Packaging Products and Services Market by Type of Therapy, Package Engineering Design, Scale of Operation and Geography : Industry Trends and Global Forecasts, 2021-2030" - https://www.reportlinker.com/p06130493/?utm_source=GNW Presently, more than 30 cell-based therapy products have received marketing approvals across various geographies, while more than 1,000 therapeutic candidates are under clinical evaluation. Considering the lucrative investment opportunity within this domain, several investors are actively supporting ongoing / future product development programs in this upcoming field of pharmacological interventions. In fact, there has been a 240% increase in investment activity, in terms of capital amount invested, between 2019 and 2020. However, the ultimate success of cell therapies is dependent on the safe and timely delivery of viable doses of therapeutic cells to the right patient. In this context, it is worth highlighting that drug product manufacturing, packaging and logistics operations involving cell therapies are both complex and challenging. Moreover, in order to ensure the stability of such products across the supply chain, specific temperature conditions (cryogenic, ambient or refrigerated) are required, in addition to specialized packaging material. Considering that most cell therapies are personalized, it is imperative that errors in labeling are avoided at all costs.

Given the need for costly and specialized equipment and the complexities associated with handling cell-based therapy products, innovators in the healthcare industry are likely to involve contract service providers for their drug product production, packaging and transportation requirements. Over time, regulators across the world, including the US Food and Drug Administration (FDA) and the International Air Transport Association (IATA), have established guidelines for the packaging, storage and transport of cell therapies. Since CMOs / CDMOs with the necessary capabilities to handle cell therapies are also likely to have established processes that comply with the current acceptable standards across different global regions, it is better for therapy developers to strategically partner with the aforementioned players instead of focusing on developing in-house capabilities. Presently, close to 60 companies claim to be offering cell therapy packaging services and associated products. In fact, it is estimated that close to 30% of drug substance and drug product related operations of advanced therapy medicinal products are already outsourced to capable CMOs / CDMOs. Further, over the last few years, several service providers have also forged strategic alliances among themselves, in order to further expand existing capabilities and augment their respective service offerings. Given the rising trend of outsourcing in the healthcare industry, and the ongoing efforts of service providers to further improve their portfolios, we believe that the cell therapy packaging products and services market is likely to evolve at a steady pace in the next few years.

SCOPE OF THE REPORT The Cell Therapy Packaging Products and Services Market by Type of Therapy (T-cell Therapies, Dendritic Cell Vaccines, Stem Cell Therapies, NK Cell Therapies and Other ATMPs), Package Engineering Design (Primary and Secondary Packaging), Scale of Operation (Clinical and Commercial) and Geography (North America, Europe, Asia Pacific and Rest of the World) , 2021-2030 report features an elaborate study of the current scenario and future opportunity within the cell therapy packaging products and services market. In addition, the report features an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain. Amongst other elements, the report includes: An overview of the current market landscape of cell therapy packaging services providers, including a detailed analysis based on their year of establishment, company size, location of headquarters, package engineering design (primary and secondary), type of packaging (active and passive), packaging material (dry ice and liquid nitrogen), type of passive system used, temperature ranges supported, type of cells handled and additional services offered. A detailed assessment of the current market landscape of cell therapy packaging products, providing information on type of packaging container (bags, vials, shipping container and others), container fabrication material (plastic and others), storage temperature conditions, type of cells packed and usability of containers. In addition, the chapter highlights analysis of cell therapy packaging product provider(s), based on various parameters such as year of establishment, company size and location of headquarters and key players. An in-depth competitiveness analysis of cell therapy packaging service providers based in different geographies, by taking into consideration the service strength of a company (based on its experience), packaging portfolio (package engineering design, type of packaging, type of packaging material and temperature ranges supported) service portfolio (type of additional services offered) and company size (small, mid-sized and large companies). An analysis of the various partnerships established between cell therapy packaging providers and cell therapy developers, during the period, 2016-2021. It includes a brief description of various types of partnership models (namely service alliance, technology / platform integration agreement, merger and acquisition) adopted by stakeholders engaged in this domain. It is worth mentioning that the data captured during our research was analyzed based on several parameters, such as year of partnership, type of partnership, purpose of partnership, type of service covered and geographical location of players involved in a partnership. A list of more than 250 cell therapy developers anticipated to partner with cell therapy packaging providers, which have been shortlisted on the basis of developer strength (based on company size), type of therapy and pipeline maturity (based on stage of development of drug candidate). A case study highlighting the companies that claim to have the required expertise and capabilities for development and manufacturing of cell therapies, along with the information on their year of establishment, company size, location of headquarters and location of manufacturing facility. In addition, the chapter presents analysis on type of cells manufactured, source of cells, scale of operation and manufacturing capabilities / services of the aforementioned companies. Detailed profiles of the key players offering cell therapy packaging services and products. Each profile includes a brief overview of the company, details on cell therapy service portfolio, information on cell therapy packaging product portfolio, recent developments and an informed future outlook.

One of the key objectives of the report was to estimate the existing market size and future growth potential of the cell therapy packaging providers market. We have provided informed estimates on the likely evolution of the market in the short to mid-term and long term, for the period 2021-2030. Our year-wise projections of the current and future opportunity have further been segmented across [A] type of therapy (T-cell therapies, dendritic cell vaccines, stem cell therapies, NK cell therapies and other advanced therapy medicinal products), [B] package engineering design (primary and secondary packaging), [C] scale of operation (clinical and commercial) and [D] geography (North America, Europe, Asia Pacific and Rest of the World)

All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified.

KEY QUESTIONS ANSWERED Who are the leading players offering cell therapy packaging services and products? What are the key considerations and challenges associated with cell therapy packaging? Which partnership models are commonly adopted by stakeholders engaged in this industry? Which players are likely to partner with cell therapy packaging service and product providers? What factors are likely to influence the evolution of the cell therapy packaging service providers market? What are the likely future trends in cell therapy packaging services market? How is the current and future opportunity likely to be distributed across key market segments?

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary research. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been validated from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views

While the focus has been on forecasting the market till 2030, the report also provides our independent views on various non-commercial trends emerging in this industry. The opinions are solely based on our knowledge, research and understanding of the relevant market trends gathered from various secondary sources of information.

CHAPTER OUTLINES Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the cell-based therapy manufacturing market and its likely evolution in the short to mid-term, and long term.

Chapter 3 provides a general introduction to the pharmaceutical filling and packaging, including information on types of pharmaceutical packaging. The chapter lays emphasis on cell therapy packaging, primary and secondary packaging of cell therapies. In addition, it discusses the key considerations and challenges associated with cell therapy packaging.

Chapter 4 features an overview of the current market landscape of cell therapy packaging services providers, including a detailed analysis on the year of establishment, company size and location of headquarters. In addition, the chapter highlights the analysis on the package engineering design (primary and secondary), type of packaging (active and passive), packaging material (dry ice and liquid nitrogen) and type of passive system used. Further, the chapter also presents analysis on temperature ranges supported, type of cells handled and additional services offered.

Chapter 5 fpresents a detailed assessment of the current market landscape of cell therapy packaging products, providing information on type of packaging container (bags, vials, shipping container and others), container fabrication material (plastic and others), storage temperature conditions, type of cells packed and usability of containers. In addition, the chapter highlights analysis of cell therapy packaging product provider(s), based on various parameters such as year of establishment, company size and location of headquarters and key players.

Chapter 6 includes profiles of the key players offering cell therapy packaging services and products. Each profile includes a brief overview of the company, details on cell therapy service portfolio, information on cell therapy packaging product portfolio, recent developments and an informed future outlook.

Chapter 7 presents in-depth competitiveness analysis of cell therapy packaging service providers based in different geographies, by taking into consideration the service strength of a company (based on its experience), packaging portfolio (package engineering design, type of packaging, type of packaging material and temperature ranges supported) service portfolio (type of additional services offered) and company size (small, mid-sized and large companies).

Chapter 8 provides an analysis of the various partnerships established between cell therapy packaging providers and cell therapy developers, during the period, 2016-2021. It includes a brief description of various types of partnership models (namely service alliance, technology / platform integration agreement, merger and acquisition) adopted by stakeholders engaged in this domain. It is worth mentioning that the data captured during our research was analyzed based on several parameters, such as year of partnership, type of partnership, purpose of partnership, type of service covered and geographical location of players involved in a partnership.

Chapter 9 presents a list of more than 250 cell therapy developers anticipated to partner with cell therapy packaging providers, which have been shortlisted on the basis of developer strength (based on company size), type of therapy and pipeline maturity (based on stage of development of drug candidate).

Chapter 10 features a case study highlighting the companies that claim to have the required expertise and capabilities for development and manufacturing of cell therapies, along with the information on their year of establishment, company size, location of headquarters and location of manufacturing facility. In addition, the chapter presents analysis on type of cells manufactured, source of cells, scale of operation and manufacturing capabilities / services of the aforementioned companies.

Chapter 11 presents a detailed market forecast analysis, providing insights on the likely growth of continuous manufacturing equipment providers market for the period 2021-2030. In addition, the chapter presents the likely distribution of the projected future opportunity based on type of therapy (T-cell therapies, dendritic cell vaccines, stem cell therapies, NK cell therapies and other advanced therapy medicinal products), package engineering design (primary and secondary packaging), scale of operation (clinical and commercial) and geography (North America, Europe, Asia Pacific and Rest of the World).

Chapter 12 is a summary of the overall report, which presents the insights on the contemporary market trends and the likely evolution of cell therapy products and services market.

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix that provides the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06130493/?utm_source=GNW

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Cell Therapy Packaging Products and Services Market by Type of Therapy, Package Engineering Design, Scale of Operation and Geography : Industry Trends...

Recommendation and review posted by Bethany Smith

Senescent immune system cells are potentially among the most harmful of all senescent cells because they spread tissue damage and rapid aging across other body organs and systems. That is what a team of NIA-supported scientists at the University of Minnesota Medical School discovered through research using a mouse model that accelerated immune system aging by hindering DNA repair. The team recently published these findings in Nature.

Cellular senescence is defined as a condition in which a cell no longer has the ability to proliferate. These damaged cells resist the bodys usual system of disposal and then linger, excreting chemicals that spread inflammation and damage to neighboring normal cells.

For this study, the team made a cell-specific knockout of the gene Ercc1, which controls a protein crucial for DNA repair. Ercc1 was removed in blood-based young stem cells that normally develop into white blood cells cells important for immunity but the gene was expressed normally in all other tissues. This enabled the research team to understand whether senescence in the immune system affects other cells in the body. The engineered mice seemed healthy up until their adulthood (around three months) but then aged rapidly. At age five months, they biologically resembled 2-year-old mice, which is approximately equivalent to an 80-year-old human.

The prematurely older mice had a host of age-related conditions such as osteoporosis; visual and hearing impairment; and high blood pressure, even though the change was limited to cells of the immune system. The senescent immune system cells also spread age-related damage to other organs and tissues in the body, including the liver, lungs, and kidneys. Without the Ercc1 gene, the mice had lost much of their ability to repair DNA in these immune cells and thus experienced a build-up of inflammation and damage in other tissues.

The scientists saw this rapid aging and spread of damage throughout the body as evidence that senescent immune system cells are potentially among the most dangerous of all senescent cell types in the aging body. Because immune cells circulate throughout the body, when they become senescent, they can easily expose a wider range of organs and tissues to inflammation and other damaging factors, unlike more stationary senescent cells such as those in the skin.

The team also studied and confirmed some mechanisms that contribute to senescence in the immune system. First, they showed that senescent immune cells trigger and drive senescence elsewhere in the body by observing senescence triggered across systems in young mice after transplanting spleen cells from old mice into them. Next, they observed that when immune cells from young healthy mice were transplanted into older mice, senescence was reduced, providing further evidence that old immune cells lose function. The scientists also used the drug rapamycin, which tamps down the inflammatory secretions from senescent cells, to show that reducing senescence improved immune function.

While the field of senescence is still very far from any reliable application for humans, the investigators aim to pursue follow-up efforts to pinpoint a precise type of senolytic a drug that selectively clears senescent cells from the body to target reducing immune system senescence as a potential future intervention to aid healthy aging. They hope to conduct additional studies in this realm to find new immune system biomarkers to help gauge which people are at the highest risk for senescence-related tissue damage and faster aging, and thus would be candidates to benefit from senolytic therapies.

This work was supported by NIH grants P01 AG043376, RO1 AG063543, R56 AG059676, U19 AG056278, P01 AG062413, R56 AG058543 and R01 AG044376.

Reference: Yousefzadeh, Matthew J et al. An aged immune system drives senescence and ageing of solid organs. Nature vol. 594,7861 (2021): 100-105. doi:10.1038/s41586-021-03547-7

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Senescent immune cells spread damage throughout the aging body - National Institute on Aging

Recommendation and review posted by Bethany Smith

Moms IL-6 rewires babys gut immunity

Most infections that occur during pregnancy are mild and transient. However, whether such pathogen encounters can shape the long-term trajectory of the offsprings immune system remains unclear. Lim et al. infected pregnant mice with the common food-borne pathogen Yersinia pseudotuberculosis (YopM) (see the Perspective by Amir and Zeng). Although the infection was maternally restricted and short-lived, the offspring harbored greater numbers of intestinal T helper 17 cells into adulthood. Interleukin-6 (IL-6) mediated this tissue-restricted effect by acting on the fetal intestinal epithelium during development. Although offspring from mothers infected with YopM or injected with IL-6 showed enhanced resistance to oral infection with Salmonella Typhimurium, they also exhibited higher susceptibility toward enteric inflammatory disease.

Science, abf3002, this issue p. eabf3002; see also abl3631, p. 967

One fundamental property of the immune system is its ability to develop memory of previous encounters, resulting in enhanced responsiveness to subsequent challenges. This phenomenon includes not only the adaptive immune system but also innate cells and tissue stem cells. The concept of host imprinting by infection leading to altered responses to subsequent challenge is of particular interest in the context of pregnancy, which represents a fundamental developmental window for the immune system.

Most infections encountered by mammals, including those experienced during pregnancy, are mild and transient. How these infections shape offspring tissue immunity and tissue predisposition to inflammatory disorders in the long term remains to be addressed.

Infection of timed-pregnant dams (day 10.5) with an attenuated strain of the food-borne pathogen Yersinia pseudotuberculosis (yopM) was transient and maternally restricted. Adult offspring of previously infected dams harbored a higher number of T helper 17 (TH17) cells but no other cell subsets in the small and large intestinal lamina propria. No changes were observed at other barrier tissues. Transfer of transgenic T cells specific for a commensal antigen revealed that maternal infection affected the offspring intestinal milieu in a manner that enhanced TH17 cell reactivity toward the microbiota. Cross-fostering experiments demonstrated that the increased TH17 cells resulting from maternal infection was imprinted in utero. Among various inflammatory mediators, the cytokine interleukin-6 (IL-6) was significantly increased in the serum of dams infected with yopM. Injection of IL-6 alone to pregnant dams significantly increased TH17 cell numbers within the guts of offspring. Conversely, blockade of IL-6 in infected dams prevented this increase in offspring. Furthermore, injection of IL-6 to germ-free pregnant dams and conventionalization of their offspring after weaning revealed that prenatal establishment and postnatal maintenance of IL-6mediated tissue imprinting are independent of the maternal microbiota but allow the offspring to mount enhanced TH17 cell responses to postnatal microbiota exposure.

IL6RA was expressed in all fetal intestinal epithelial cells, and specific deletion of IL6RA from epithelial cells significantly reduced Th17 responses within the gut of offspring from IL-6exposed dams. Using complementary approaches, including assay for transposase-accessible chromatin with high-throughput sequencing (ATAC-seq) and single-cell RNA sequencing, we found that increased IL-6 during pregnancy had immediate effects (on fetal cells) and long-term effects (in the adult offspring) on epithelial stem cell chromatin accessibility and downstream epithelial cell function, particularly increased expression of genes encoding for antigen presentation machinery and antimicrobial peptides.

Altered epithelial activation status suggested the possibility that this phenomenon may be associated with enhanced antimicrobial defense. To address this, we used an acute model of oral infection with Salmonella Typhimurium. Offspring from dams previously infected with yopM or injected with IL-6 during pregnancy developed enhanced resistance to Salmonella oral infection and dissemination. No differences were observed in controlling cutaneous Candida albicans infection, further supporting the idea that maternal imprinting of offspring is restricted to the gut compartment. However, enhanced exposure to IL-6 during pregnancy was associated with enhanced gut pathology in the context of nave T cell transfer and dextran sulfate sodiummediated colitis.

Our work proposes that a transient, mild infection encountered during prenatal development can impose lasting alterations to gut epithelial stem cells, resulting in an altered threshold of activation and enhanced resistance to gut infection. The impact of maternal infection was tissue specific and predominantly mediated by a single cytokine, IL-6, acting on epithelial stem cells during fetal development. Although this phenomenon can be coopted by the fetus to develop optimal immune fitness, altered offspring immunity imposed by maternal infection comes at the cost of enhanced susceptibility to mucosal inflammation.

The direct response of fetal intestinal epithelial cells to IL-6 during maternal infection confers an enduring epigenetic memory to adult intestinal epithelial stem cells. As such, offspring epithelial cells exhibit enhanced reactivity toward the microbiota and heightened ability to control oral infection. However, these responses come at the cost of greater predisposition to gut inflammation.

The immune system has evolved in the face of microbial exposure. How maternal infection experienced at distinct developmental stages shapes the offspring immune system remains poorly understood. Here, we show that during pregnancy, maternally restricted infection can have permanent and tissue-specific impacts on offspring immunity. Mechanistically, maternal interleukin-6 produced in response to infection can directly impose epigenetic changes on fetal intestinal epithelial stem cells, leading to long-lasting impacts on intestinal immune homeostasis. As a result, offspring of previously infected dams develop enhanced protective immunity to gut infection and increased inflammation in the context of colitis. Thus, maternal infection can be coopted by the fetus to promote long-term, tissue-specific fitness, a phenomenon that may come at the cost of predisposition to inflammatory disorders.

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Prenatal maternal infection promotes tissue-specific immunity and inflammation in offspring - Science Magazine

Recommendation and review posted by Bethany Smith

A patient recently came in to our dermatology clinic with a rash and a story similar to so many others. He had been out camping with friends a few days earlier and helped carry some logs to stoke the fire. Little did he know he was going to pay for lending a helping hand. A couple days later, red patches appeared on his forearms and chest, which soon began to itch miserably and form water blisters.

If you have ever spent any time outdoors in the woods, working in the yard, even at the edges of a playground maybe youve experienced something similar after encountering poison ivy. Its not easy to forget.

Poison ivy is found everywhere in the continental U.S., mostly in Eastern and Midwestern states. Unfortunately for us humans, it is a hardy plant that can grow under many different conditions. Its favorite places are in wooded areas, gardens and roadsides with partial shade or full sunlight.

And despite being a nuisance to people, poison ivy is an important member of the ecosystem. Its leaves, stems and berries are food for animals, and its vines can be shelter for small animals like toads and mice, even helping them climb trees. Climate change is turning out to benefit poison ivy, allowing for larger and more irritating plants.

You can usually spot poison ivy by its infamous three dull or glossy green leaves coming off a red stem. Sometimes there are flowers or fruits coming off the end of a branch.

Despite its name, poison ivy is not poisonous. It carries an oily sap on its leaves and stems called urushiol, which is irritating to most peoples skin. In fact, 85% to 90% of people are allergic to poison ivys urushiol to some degree, while the rest lack sensitivity to this oil. You can occasionally see the urushiol oil as black spots on poison ivy leaves. Urushiol is what gives poison oak and poison sumac their evil power, too.

Touching poison ivy directly is obviously a bad idea. You can even get into trouble by touching clothing, pets or anything else that has brushed against the plant and picked up some of the urushiol. If a contaminated object isnt cleaned, the urushiol will remain lying in wait it can still cause a rash after hours, days or even years. Another danger is smoke from burning poison ivy, which can also affect your skin, as well as your nose, mouth, windpipe and lungs if you breathe it in.

Poison ivys rash can come in many forms, from small, red bumps to blisters or red patches. Whichever way it shows up, it is almost always mindbogglingly itchy.

When you get poisoned, you wont know right away. It can take anywhere from four hours to 10 days for the rash to appear, depending on how much urushiol gets on your skin, how sensitive you are to it and how many times you have been exposed to poison ivy previously.

Between exposure and itchy anguish, your body goes through a complex identification and reaction process. When the oil gets into your skin, your immune systems sensor cells recognize urushiol as foreign to your body. These sensor cells then call in protector cells to the area, warning them of the invasion. The protector cells defend your body against the intruder by attacking the urushiol in the skin. Unfortunately, some of your bodys normal skin cells are casualties of this war, which is what leads to the itchiness and swelling of a poison ivy rash.

Your protector cells will then sit near the skin for many years and stand guard for urushiol if it ever shows up again. If it does, they remember having encountered this bad guy before, and their response is often faster and more powerful than the first time.

This rash is a type of allergic contact dermatitis in the same family as the rashes some people get from wearing jewelry or metal belt buckles or from using certain fragrances or cosmetics.

The saying leaves of three; leave them be highlights the best strategy to prevent poison ivy: avoidance. But if you do happen to come into contact with poison ivy, the first step should always be to remove and wash any clothing that has touched the plant. Gently but thoroughly wash your skin immediately with soap and water. It can also help to clean under your fingernails and cut your nails short to prevent the urushiol from spreading if you scratch your skin.

Allergic contact dermatitis from poison ivy almost always results in a rash that usually lasts two to three weeks before it completely goes away.

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It will eventually clear up on its own, but you can try some over-the-counter and home remedies to keep the itchiness and spread of the rash at bay. The blisters that form are not infected and do not normally require antibiotics. If you scratch though and it can be very hard to resist open skin can get infected.

To reduce itchiness, cool, wet compresses can help, as can a soak in a cool bath with baking soda or oatmeal bath products. Calamine lotions or creams containing menthol can also cut the itch a bit. Over-the-counter cortisone cream or ointment can be used for the first several days after contact with poison ivy to quiet down your bodys reaction and keep the rash from getting severe. Taking antihistamines like diphenhydramine at night can slightly reduce itchiness and it has the benefit of helping you sleep better.

Seeing your doctor usually is not necessary for a poison ivy rash unless it spreads over large areas, becomes infected, lasts more than three weeks or is a rare extreme case that affects your breathing.

The best offense is a good defense. When youre in the great outdoors, be careful what you touch and, when in doubt, if it has leaves of three, leave them be.

Read more:
Poison ivy can work itchy evil on your skin here's how - The Conversation US

Recommendation and review posted by Bethany Smith

Work-from-home, double taps, left swipes, binge-watch and reel challenges are a few of the many Internet jargons which have become our constant companions. Thanks to the pandemic and restrictions on moving freely, our work and playtime are both equal to screen time. And as satisfying and relaxing as those cat jade rolling videos might be, increasing your screen time is doing no favours to your skin.

Unlike UV rays which are invisible to the eye, High Energy Visible Light (HEV) or blue light emitted from electronic screens is visible to us. It increases the oxidative damage to our skin and is said to make it age faster. Melasma, skin discolouration, pigmentation, collagen breakdown and wrinkle formation are a few of the skin issues that might be a result of or worsen due to blue light, which is found in sun rays too. While we are yet to know the extent to which blue light from screens can affect our skin due to free radical damage, we do know that using antioxidant-rich products can help you tackle this concern. So if your skincare already includes antioxidants such as vitamin C and E, you are in the zone. And if you use mineral sunscreens that contain zinc oxide and titanium dioxide, its doing double the work by blocking UV rays and blue light to an extent. So if you use vitamin C, ferulic acid and top it with sunscreen, your skin is pretty walled up against all kinds of light damage.

Switching your phone to night mode will help your case, but including these or similar products in your routine will only do more good and no harm.

More here:
7 products that will arm your skin against blue light damage - VOGUE India

Recommendation and review posted by Bethany Smith

Nature of Things launched the Elemental Skincare collection in April, which features a complete ... [+] four-step facial routine.

As the summer season winds down, now is the perfect time to start thinking about how to streamline your skincare routine to keep that sun-kissed glow and stay protected from the elements all year long.

I might be biased (and, yes, I test cannabis products as part of my job) but over the past few years, Ive transitioned to an all-CBD-based face and body regimen (with the exception of Biologique RechercheLotion P50 and EltaMD sunscreen). The result? My clearest and healthiest complexion to date.

With BDSAs recent trend report forecasting the CBD beauty segment of the market to reach $720 million this year alone a 60% increase over 2020 and representing just 10% of the total CBD market the number of new brands and product launches to consider buying often at a high price tag is overwhelming.

Whether you are looking to make a complete switch to CBD or want to try just one step at a time, here are the nine best cannabis beauty launches of 2021 (so far) to help narrow your search each tried-and-tested in the challenging and drying environment of the high alpine.

*Note: This list is in alphabetical order, not ranked and will continue to get updated with new product launches through December 2021.

Vitamin C + Squalane + CBD Skin Elixir

This antioxidant-rich formula combines three superstar ingredients vitamin C, squalane and CBD to create a natural skin brightener, which targets dark spots and reduces signs of aging. Best used after moisturizer and under makeup in your morning routine, Botanika Lifes newest serum is infused with 1,000 milligrams of full-spectrum CBD extract, which helps with hydration and restore natural radiance. And dont forget to layer with sunscreen after you apply to improve sun protection.$95, botanikalife.com

Look Alive Face Moisturizer

For celebrity co-founder Kristen Bell, the havoc wreaked on her skin from the normal stresses of the day was the inspiration in creating Happy Dances debut face moisturizer. Formulated as a first line of defense barrier, this whipped, hydrating creme is light on skin, but heavy on hydration. Infused with 150 milligrams of CBD extract, its also powered by avocado oil, bisabolol with a ginger blend andfour types of hyaluronic acid. Plus, its been ophthalmologist-tested (for safe use around the eyes) and available at a more accessible price point than most CBD-based moisturizers on the market.$29, doahappydance.com

Bleu Body Wax

Khus + Khus founder Kristi Blustein is an Ayurvedic specialist, aromatherapist and herbalist experience that makes her modern herbal fusion mission stand apart among the crowded CBD beauty product space.Released earlier this year, the Bleu Body Wax is a concentrated pomade thats solid at room temperature and when warmed up in your hands, liquifies for smooth application. Each jar is packed with 225 milligrams of broad-spectrum CBD complemented by coconut, baobab seed, Marla and beeswax with essential oils like tiara flower and blue yarrow to perfume the body naturally. $77, khus-khus.com

The Rose Absolute Oil

So its not technically a CBD product pick, but Lab to Beauty turned to another cannabis compound for a second skincare collection. CBG (cannabigerol) is just one of more than 120 identified cannabinoid compounds found in the plant genus. It is known to calm inflammation, reduce blemishes, purify pores, balance sebum production and assist with cellular turnover. Concocted with Bulgarian rose extract, golden jojoba oil, moringa, immortelle and rosehip oil, this luxurious and healing formula works to fight aging, improve elasticity and deliver a youthful plump. Use The Rose Absolute Oil nightly after cleansing and toning or under your favorite foundation to boost brightness. $145, labtobeauty.com

Bump & Smooth CBD Body Serum

Lord Jones recent release is the answer to one of the most common skin issues: those mysterious mini-bumps and rough patches often inflicted by too much time in the sun. This potent resurfacing serum delivers a non-abrasive chemical exfoliation experience that reduces bumpiness and reveals smoother, brighter-looking skin thanks to 200 milligrams of full-spectrum CBD extract blended with bisabolol, hyaluronic acid, ceramides and squalane. Whether you suffer from Keratosis Pilaris (KP) or are just looking for a refresh to dull skin, Lord Jones Bump & Smooth CBD Body Serum gently removes dead cells to reveal whats underneath. $60, lordjones.com

Facial Essence

Beloved for its body care and bath immersion portfolio of products, Nature of Things introduced a skincare line earlier this year featuring a cleanser, toner, moisturizer and mask (face stone optional). While the entire four-step routine is remarkable, the Balancing Facial Essence has remained in my medicine cabinet because of its soothing, hydrating and dewy result from pure ingredient extracts like broad-spectrum CBD, Swiss apple stem cells, Korean kombu algae and French thermal water. Bonus benefits include new skin cell growth, refined pores and free radical protection. $95, natureofthings.com

Beyond Body Oil

Supercharged with 600 milligrams of hemp CBD oil per generous-sized bottle, Primas latest addition to its lineup goes way beyond your average body oil. Plant actives including omegas, phenols, antioxidants and fatty acids are meticulously combined with magnesium and helichrysum in this soothing, therapeutic formula, which actually repairs and restores the skin while also improving tone and texture. Pro-tip: Keep it in your shower for application to breathe in the aromatic blend of clove, geranium and citrus and lock in most moisture just before getting out. $56, prima.co

Hydrating Petal Cream

Best-selling CBD beauty brand Saint Jane has done it again with its first-ever face cream (previous award-winning releases in the category include The C-Drops, Bright Repair Eye Cream and Luxury Beauty Serum). Founder Casey Georgeson spent more than a year searching for the flower thats best for skin hydration and hibiscus, known as natures Botox, was ultimately selected for its efficacy in firming and restoring elasticity to the skin. Blended with 1% pure hyaluronic acid, the lightweight Hydrating Petal Cream is a joy to apply, absorbing quickly into the skin and leaving the face with a dewy finish that lasts all day. $68, saintjanebeauty.com

Glow Drops

They say beauty comes from within, so incorporating a CBD-based tincture into your regimen is a secret weapon in supercharging your skin. As the first-to-market beauty-focused elixir, Undefined Beautys Glow Drops can also be mixed into any moisturizer to achieve an extra shine. Paired with adaptogens ranging from mucuna (mood-boosting) and cordyceps (anti-inflammatory) to guava leaf (immunity-boosting) and astaxanthin (antioxidant), the tasty tangerine formula is an easy way to simplify your daily dose whether taken under the tongue or added to a beverage. Each full-size bottle includes over 600 milligrams of CBD, CBD and CBC (cannabichromene) sourced from a certified-organic, female-founded regenerative hemp farm. $48, undefinedco.com

Read the original post:
The Best New CBD Skincare Products Of 2021 (So Far) - Forbes

Recommendation and review posted by Bethany Smith

One of the key factors fueling the growth of the global hormone replacement therapy market is increasing acquisitions. Pfizer Inc., a pharmaceutical firm headquartered in the United States, combined with OPKO Health Inc., a business based in the U.S., in 2014 to create a long-acting growth hormone (hGH-CTP) and novel therapies for growth hormone deficient individuals. hGH-CTP is more convenient since patients just require one injection each week instead of daily dosages. In the U.S. and Europe, hGH-CTP has been designated as an orphan medication for children and adults with growth hormone deficiencies.

The expansion of the global hormone replacement therapy market is projected to be aided by a strong pipeline. In 2017, Novo Nordisk A/S, a Danish firm, completed phase 3 clinical studies for Somapacitan. This medication is used to treat adult testosterone deficiency.

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Major Company Profiles Covered in This Report:Novartis AG, Abbott Laboratories, Mylan N.V., Merck KgaA, Bayer AG, Pfizer Inc., Novo Nordisk A/S, QuatRx Pharmaceuticals, Teva Pharmaceutical Industries Ltd., Amgen, Inc., and Eli Lilly and Company.

The market for hormone replacement treatment is projected to expand due to the rising prevalence of various chronic diseases.

The market for hormone replacement treatment is projected to expand due to the rising prevalence of hypogonadism in adult males throughout the world. Hypogonadism affects 2.1 percent to 12.8 percent of middle-aged men, according to the European Association of Urologys 2016 study. In Europe, the prevalence of low testosterone and hypogonadism symptoms in males aged 40 to 79 ranges from 2.1 percent to 5.7 percent.

The expansion of the global hormone replacement therapy market is projected to be aided by easier access and government assistance for research and development efforts. NGOs such as the National Gaucher Society provide financial assistance to patients who require expensive insulin replacement treatment.

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Companies are concentrating their efforts on creating generic versions of numerous medications for the treatment of diseases that are more common in women, such as hypothyroidism. On July 24, 2017, Teva Pharmaceutical Industries Ltd. introduced a generic version of Vagifem, 10 mcg, in the United States. Estradiol vaginal inserts are a kind of oestrogen used to treat atrophic vaginitis caused by menopause.

Furthermore, Mylan N.V. received FDA clearance for its Abbreviated New Medication Application (ANDA) for Estradiol Vaginal Cream USP, 0.01 percent on December 29, 2017, and therefore marketed the drug in the U.S. This cream is the first generic alternative to Allergans Estrace Cream, which is used to treat vulvar and vaginal atrophy.

Mylan is one of the few firms that sells Estradiol in cream, gel, transdermal patch, and tablet form. This will benefit both healthcare personnel and patients, as well as ensuring the businesss long-term viability.

Major companies contributing in the global hormone replacement therapy market are Pfizer Inc., QuatRx Pharmaceuticals, Mylan N.V., Abbott Laboratories, Amgen, Inc., Novartis AG, Eli Lilly and Company, Bayer AG, Merck KgaA, Teva Pharmaceutical Industries Ltd., and Novo Nordisk A/S.

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Recommendation and review posted by Bethany Smith